Comprehensive research guides that help patients and families walk into medical conversations better prepared.
100 research guides available — 130+ more in development — free, evidence-based, regularly updated
Each guide covers what the disease is, what treatment options exist, which questions to ask, and where to find the best care — organized by where you are in the journey.
Diagnosis, dry-eye and dry-mouth management, systemic treatment options, emerging therapies, clinical trials, and specialty centers.
Available NowHSV-1 (oral), HSV-2 (genital), and herpes zoster (shingles/VZV) — diagnosis, antiviral and suppressive therapy, complications, pregnancy, and emerging treatments.
Available NowDiagnosis, staging, surgery, adjuvant chemotherapy (IDEA framework), molecular testing (dMMR/MSI, RAS, BRAF V600E, HER2), Lynch syndrome, targeted therapies, ctDNA, clinical trials, and major centers.
Available NowStandard treatment, clinical trials, repurposed therapies, regional medical centers, and what to do at every stage from diagnosis through recurrence.
Available NowBiomarker testing, targeted therapies (EGFR, ALK, KRAS G12C), immunotherapy, surgical advances, radiation, clinical trials, and comprehensive cancer centers.
Available NowRisk stratification, induction therapy, stem cell transplant, maintenance, CAR-T and bispecific antibodies, bone health, and smoldering myeloma monitoring.
Available Now12+ NHL subtypes, standard treatments, CAR-T and bispecific antibodies, repurposed drugs, international approaches, diet and metabolic strategy, and high-volume centers.
Available NowUnderstanding PDAC and what resectable/borderline/advanced mean, warning signs & family risk, chemo options explained (incl. NALIRIFOX), the KRAS breakthrough & trials, choosing a high-volume center & Whipple recovery, enzymes & nutrition, pain & palliative care, and an honest word on hope.
Available NowRisk stratification, active surveillance, surgery, radiation, ADT & intensification, mCRPC, PSMA-targeted therapy, genomic testing, and clinical trials.
Available NowAll four subtypes (HR+/HER2-, HER2+, HER2-low, triple-negative): diagnosis and staging, genomic testing (BRCA, Oncotype, PD-L1, ESR1, PIK3CA), surgery and radiation, endocrine and CDK4/6 therapy, HER2-targeted agents, ADCs, immunotherapy, metastatic care, trials, and major centers.
Available NowThyroid nodule evaluation, molecular testing (Afirma, ThyroSeq), Bethesda classification, surgery options, radioactive iodine therapy, TSH suppression, targeted therapies for RET/BRAF/NTRK mutations, active surveillance, medullary and anaplastic management, and clinical trials.
Available Now~6K new/yr US. Blinatumomab moving into frontline consolidation (E1910); CAR-T (brexucabtagene, tisagenlecleucel); MRD-guided therapy.
Available Now~20K new/yr; ~11K deaths. Menin inhibitor revumenib (Revuforj, FDA 2024) for KMT2A/NPM1; expanding IDH1/2 and FLT3 inhibitors; oral azacitidine maintenance.
Available Now~1-2 per million/yr. Mitotane plus chemotherapy standard; immunotherapy and targeted trials; hormone-excess management.
Available Now~10K new/yr US. Immunotherapy in advanced disease; HPV-driven prevention and high-resolution-anoscopy screening (ANCHOR).
Available Now~83K new/yr US. Enfortumab vedotin + pembrolizumab (EV-302) transformed first-line metastatic; nadofaragene and N-803 for BCG-unresponsive NMIBC.
Available Now~5-6K new/yr US (all types). Siltuximab for idiopathic multicentric disease; sirolimus and JAK-pathway trials; CDCN-driven research.
Available Now~14K new/yr US. Pembrolizumab + chemoradiation (KEYNOTE-A18); tisotumab vedotin for recurrent disease; HPV-driven prevention.
Available Now~8-12K new/yr US. FGFR2 inhibitors (pemigatinib, futibatinib), IDH1 inhibitor ivosidenib, durvalumab+chemo (TOPAZ-1) first-line.
Available Now~21K new/yr; ~200K prevalence. Fixed-duration BTK+BCL2 (venetoclax-based) combinations; pirtobrutinib for BTK-resistant disease.
~3K new/yr US. Mogamulizumab; expanding skin-directed and systemic options; stage-driven therapy.
~5-6 per million/yr. Nirogacestat (Ogsiveo, 2023) — first FDA-approved therapy; gamma-secretase inhibition.
Available Now~25K new/yr US. Bispecifics epcoritamab and glofitamab; pola-R-CHP first-line (POLARIX); CAR-T moved to second line.
~300-400 new/yr US. Dordaviprone (Modeyso) accelerated approval (2025) for H3 K27M-mutant disease — first targeted option.
Adeno vs squamous, Barrett’s & early warning signs, surgery and recovery, chemo/radiation, immunotherapy explained, fertility & sexual health, caregiving, trials, and a hopeful, plain-language path through every stage.
~200 new/yr US. Refined multimodal therapy; trials of targeted agents; survivorship-focused care.
~27K new/yr US. Zolbetuximab (Vyloy) for Claudin 18.2-positive tumors; trastuzumab deruxtecan; IO by PD-L1 status.
Available Now~71K new/yr US. Cancer by subsite (mouth, throat, voice box, nasopharynx), HPV-related throat cancer, surgery (incl. TORS), radiation and chemoradiation, perioperative pembrolizumab (KEYNOTE-689), immunotherapy for advanced disease, organ preservation, and protecting voice and swallowing.
Available Now~42K new/yr US. Atezolizumab+bevacizumab and durvalumab+tremelimumab first-line; IMbrave050 adjuvant data; expanding earlier-stage IO.
~5-6K new/yr US. Vorasidenib (Voranigo, FDA 2024) — first targeted therapy for grade 2 IDH-mutant glioma (INDIGO trial).
~100K new/yr US. Tumor-infiltrating lymphocyte therapy lifileucel (Amtagvi, 2024); adjuvant IO; mRNA-4157 personalized vaccine (with pembrolizumab) phase 3.
~3K new/yr US. Avelumab and pembrolizumab — checkpoint inhibitors transformed this aggressive skin cancer; neoadjuvant IO trials.
~3K new/yr US. Nivolumab+ipilimumab first-line (CheckMate-743); perioperative IO trials; ADCs in development.
~700-800 new/yr US. Anti-GD2 immunotherapy (dinutuximab, naxitamab) standard in high-risk disease; eflornithine maintenance.
~1K new/yr US. Limb-salvage surgery advances; immunotherapy and targeted trials; metastatic-disease research.
~20K new/yr; ~12K deaths. PARP-inhibitor maintenance (HRD-guided); mirvetuximab soravtansine for folate-receptor-alpha-positive disease.
Available Now~82K new/yr US. IO-TKI combinations standard; belzutifan (HIF-2alpha inhibitor) for VHL and advanced disease; adjuvant pembrolizumab.
~200-300 new/yr US. Intra-arterial and intravitreal chemotherapy enable eye preservation; genetic (RB1) testing and counseling.
Chemo-immunotherapy for both stages, consolidation durvalumab (ADRIATIC), brain-protecting radiation, lurbinectedin maintenance, and the DLL3-targeted bispecific tarlatamab (Imdelltra, full FDA approval Nov 2025) for relapse — with paraneoplastic-syndrome care, side-effect management, and survivorship.
~13K new/yr US. Subtype-specific advances; nirogacestat for desmoids; afamitresgene autoleucel (T-cell therapy) for synovial sarcoma (2024).
~400-500 new/yr US. Refined multimodal therapy; immunotherapy use tempered by autoimmune risk; paraneoplastic awareness.
~2.5K new/yr US. Tebentafusp (Kimmtrak) — first therapy to extend overall survival in metastatic disease (HLA-A*02:01 patients).
Degenerative spine disease, pinched nerves (radiculopathy), spinal-cord compression (cervical myelopathy), and lumbar spinal stenosis — how to tell them apart, the emergency warning signs (cauda equina), non-surgical care vs. when decompression surgery helps, why timing matters for cord compression, the treatable-compression-vs-ALS question, and pregnancy considerations.
Available NowUnderstanding AD stages, amyloid-targeting therapies (lecanemab, donanemab), cognitive support, caregiver guidance, clinical trials, and specialty memory centers.
Available NowGold Coast criteria, genetic testing (SOD1/C9orf72), disease-modifying medicines, multidisciplinary ALS clinic care, breathing support, communication tools, caregiver guidance, and advance care planning.
Available NowAddendum covering MSA, Dementia with Lewy Bodies (DLB), and Pure Autonomic Failure (PAF). Differential diagnosis, MDS 2022 MSA criteria, autonomic management, and supplement evidence grades.
Available NowFcRn blockers, complement inhibitors, thymectomy, crisis management, medication interactions, pregnancy considerations, and specialist referral.
Available NowStage-based guide covering medications, exercise programs, DBS, emerging therapies, clinical trials, and specialty centers with a dedicated Canada tab.
~1 in 12-20K. Antisense-oligonucleotide programs targeting UBE3A in trials; symptomatic-care optimization.
~1 in 25-50K. Genetic diagnosis (NOTCH3); supportive management; emerging therapeutic research.
~1 in 200-600. Propranolol and other repurposed-drug trials; familial-form genetic testing; imaging-surveillance guidance.
The most common inherited neuropathy (~1 in 2,500). Subtypes and genetics, diagnosis, the drugs to avoid (vincristine contraindicated), telling CMT from treatable look-alikes (CIDP, hereditary ATTR amyloidosis), braces and supportive care that keep you mobile, foot care, pain, CMT in children, the CMT1A treatment trials (PXT3003), and family planning.
~20-30K US. Efgartigimod (Vyvgart) approved for CIDP (2024); maintenance immunoglobulin optimization.
~4M US with chronic migraine. CGRP monoclonal antibodies and oral gepants for prevention and acute use; growing real-world evidence.
~1 in 1K. Galcanezumab for episodic cluster; non-invasive vagus nerve stimulation; oxygen and acute-treatment optimization.
~1 in 15-16K births. Fenfluramine and cannabidiol approved; STK-001 antisense oligonucleotide and gene-therapy trials.
~1M US with refractory seizures. New antiseizure medications (cenobamate); responsive neurostimulation and laser ablation; surgical-evaluation push.
The treatable look-alikes you must not miss (dopa-responsive dystonia, Wilson disease), the types, botulinum toxin (incl. longer-acting daxibotulinumtoxinA), deep brain stimulation, pain and emotional-health care, sensory tricks, dystonic-storm warning signs, and support.
~7M US. MRI-guided focused ultrasound thalamotomy — incisionless option; expanding bilateral treatment.
~1 in 4-7K. Targeted trials (e.g., zatolmilast/BPN14770 PDE4D inhibitor) for cognition; refined behavioral care.
~5K US. Omaveloxolone (Skyclarys, 2023) — first-ever approved treatment; gene therapy and frataxin-replacement programs advancing.
Available Now~50-60K US. Progranulin-raising therapies (latozinemab) and C9orf72-targeted ASOs in trials; AC Immune and others advancing.
~1-2 per 100K/yr. IVIG and plasma exchange standard; complement inhibitor (eculizumab) and other trials for severe disease.
~10-40K US (underdiagnosed). TTR silencers patisiran, vutrisiran, eplontersen; NTLA-2001 (Nextriva) one-time CRISPR therapy advancing.
~1-5 per 100K. Gene-discovery expansion; subtype-specific trials emerging; supportive-care optimization.
~41K symptomatic US; ~200K at-risk. AMT-130 AAV gene therapy showed first-ever signal of slowed progression (2025); huntingtin-lowering ASO programs continuing.
~3 per million. Amifampridine (Firdapse/Ruzurgi); strong association with small-cell lung cancer driving cancer screening.
~100 new/yr US. Idebenone; lenadogene nolparvovec gene-therapy trials; mitochondrial-targeted approaches.
~30-50K US. Cannabidiol and fenfluramine approved; cenobamate and other agents studied; dietary and device options.
~1.4M US. Refined biomarkers (skin/CSF alpha-synuclein seed amplification); neuroleptic-sensitivity awareness; symptomatic-care advances.
Available NowThe 2nd most common — and most preventable — dementia. The types and how it differs from Alzheimer's, diagnosis, why controlling blood pressure and preventing strokes is the real treatment (SPRINT-MIND), mixed dementia, daily strategies, and resources. “What's good for the heart is good for the brain.”
Available NowThe most common substrate of dementia in older adults (Alzheimer’s + cerebrovascular). Treat both processes; anti-amyloid eligibility tempered by vascular/microbleed burden.
Available NowOne of the few potentially reversible causes of dementia. The Hakim triad (gait, thinking, bladder), diagnosis by imaging and the tap test, shunt surgery and what the 2025 PENS trial showed (gait benefit), realistic expectations, falls, and finding an experienced center.
Available NowDementia >1 year after Parkinson’s motor onset (the 1-year rule vs DLB). Rivastigmine FDA-approved; the critical neuroleptic-sensitivity warning; treating reversible decline (delirium); fluctuations, hallucinations, non-motor care, caregiver support, and the (thin) trial pipeline.
Available NowA preventable, treatable thiamine-deficiency disorder. The Wernicke emergency (urgent injected thiamine before glucose — treat on suspicion), the chronic Korsakoff amnesia and its outlook, causes (alcohol, bariatric surgery, hyperemesis), prevention, and support — with the warning signs that should prompt urgent care.
Available NowThe ANI/MND/HAD spectrum. Severe dementia is now rare with effective HIV treatment — sustained viral suppression protects the brain. Covers the many treatable causes of thinking problems, CSF viral escape, aging and the brain, daily strategies, and support.
Available NowRapidly progressive prion dementia with myoclonus and ataxia; RT-QuIC + DWI-MRI diagnosis. No disease-modifying treatment — supportive/palliative; median survival months.
~1-2 per 100K. Newly defined entity (MOGAD); rozanolixizumab and other trials; distinguished from MS and NMOSD.
~1 in 100K US (higher in East Asia). Revascularization surgery (bypass) is mainstay; refined patient selection and imaging.
The multisystem muscle disease where the heart and anesthesia — not the visible weakness — carry the biggest risk. DM1 vs DM2, why monitoring matters when you feel well, treating myotonia, the congenital form, cardiac & breathing surveillance, pregnancy and inheritance, and the RNA-targeted trials.
Available Now~150-200K US. Orexin-receptor agonists (oveporexton/TAK-861) in phase 3 — potentially disease-targeted, not just symptomatic.
~12K new/yr US; rising prevalence. Lutetium-177 dotatate (Lutathera) expanding to earlier lines; somatostatin-analog and targeted-agent advances.
A lifespan guide: the features & diagnosis, age-based surveillance, the cancer (MPNST) warning signs, children's eye monitoring (optic pathway glioma), learning & school support, treatable high blood pressure, MEK-inhibitor medicines (selumetinib, mirdametinib), trials, and family planning.
Available Now~15K US. Four approved biologics: eculizumab, satralizumab, inebilizumab, and ravulizumab (2024) — relapse prevention transformed.
Available Now~7-10M US with significant symptoms. Shift away from dopamine agonists (augmentation risk) toward alpha-2-delta ligands and iron repletion.
~6-9K US. Trofinetide (Daybue, 2023) — first-ever approved drug; gene-therapy programs (e.g., TSHA-102) in trials.
~1-5 per 100K. Troriluzole and gene-specific antisense programs in trials; expanded genetic testing.
~150K US. Microvascular decompression and modern radiosurgery/ablation; newer sodium-channel-blocker research.
~50K US. mTOR inhibitors (everolimus) for SEGAs/angiomyolipomas; cannabidiol for seizures; surveillance protocols.
Progressive-MS emphasis: diagnosis, disease-modifying therapies, BTK inhibitors, symptom management, clinical trials, and resources for patients and caregivers.
Available Now~40-50K US. Avacopan (Tavneos) — oral C5a-receptor blocker enabling major steroid reduction; rituximab maintenance optimization.
~1-2 per million/yr. IL-1 (anakinra, canakinumab) and IL-6 inhibitors; recognition of macrophage-activation-syndrome risk.
~100-200K US. Standard steroid/azathioprine plus emerging steroid-sparing strategies; refined diagnostic scoring.
~1.5-3M US (incl. non-radiographic). IL-17 inhibitors and JAK inhibitors; non-radiographic axSpA recognition; earlier diagnosis push.
~1 in 20K US (higher along Silk Road). Apremilast for oral ulcers; TNF and IL-1 inhibitors; organ-specific treatment frameworks.
~2-5 per 100K/yr (elderly). Dupilumab approved for bullous pemphigoid (2025) — first targeted biologic; steroid-sparing.
~10 per 100K. IVIG (Octagam 10%) approved for dermatomyositis; myositis-specific antibody-driven subtyping; JAK inhibitors.
~1 in 200-1K in at-risk populations. Colchicine remains first-line; IL-1 inhibitors for colchicine-resistant disease; MEFV genetic testing.
~228K US over 50. Tocilizumab steroid-sparing; new IL-6 and GM-CSF-pathway trials; fast-track diagnostic pathways.
~1-5 per 100K (likely underdiagnosed). Inebilizumab (MITIGATE trial, 2025) — first agent with a dedicated IgG4-RD program; rituximab widely used.
~5-10 per 100K over 50. No approved treatment; ulnar/finger-flexor pattern recognition; trials of myostatin and autophagy-targeted agents.
~1-5 per million/yr. Rituximab first-line (with steroids) is now standard; FcRn and other targeted agents in trials.
~1.5M US. IL-17, IL-23, and JAK inhibitors; bimekizumab; domain-based treatment selection.
~1.3M US. Diagnosis and blood tests, treat-to-target care, methotrexate and DMARDs, biologics and JAK inhibitors (with safety), pregnancy planning, and protecting your joints.
Available Now~0.4-3.1M US (wide estimates). Ianalumab, dazodalibep, and other B-cell/co-stimulation agents in phase 3 — first potential disease-modifying drugs.
~1-2 per million. No approved disease-specific drug; rituximab and immunotherapy used off-label; GAD/glycine-receptor antibody subtyping.
~100K US. Tocilizumab and nintedanib for SSc-ILD; autologous stem-cell transplant; CD19 CAR-T trials showing remission.
Rate vs. rhythm control, ablation, anticoagulation strategies, stroke prevention, lifestyle modifications, and shared decision-making.
Available NowTTR stabilizers (tafamidis, acoramidis), gene silencers, cardiac monitoring, genetic testing, transplant pathways, and multidisciplinary care.
Available NowDiagnosis, GDMT optimization, HFpEF treatment (SGLT2i, tirzepatide), ATTR-CM (tafamidis, acoramidis), devices, transplant, clinical trials, and patient resources.
Available NowSotatercept, combination therapy, risk stratification, right heart catheterization, exercise, transplant evaluation, and PH center referral.
Available NowStroke biology, prevention (Life’s Essential 8), three recovery phases, rehabilitation settings, neuromodulation, spasticity management, international approaches, and comprehensive stroke centers.
~1.5M+ US (moderate-severe). EARLY TAVR (2024) supports earlier intervention in asymptomatic severe disease; TAVR durability data maturing.
~1 in 2-5K. Genetic-testing-driven diagnosis; exercise-restriction evidence; risk-stratified ICD decisions.
~1 in 2K (higher in Asia). Risk-stratification refinements; ajmaline-challenge use; ICD and ablation decision frameworks.
Underdiagnosed subset of sarcoidosis. Advanced imaging (cardiac MRI/FDG-PET) recognition; immunosuppression plus device therapy protocols.
~20M US. Aggressive LDL lowering (statins, ezetimibe, PCSK9 inhibitors incl. once-monthly lerodalcibep, inclisiran, bempedoic acid), lipoprotein(a), colchicine for residual inflammation, blood thinners, stents vs. bypass, cardiometabolic medicines, and cardiac rehab.
~1 in 5K (largely undiagnosed). Pomalidomide (PATH-HHT trial, 2024) reduced bleeding; anti-VEGF strategies; systemic therapy emerging.
~1 in 2K. Genotype-specific management; beta-blocker selection by subtype; left cardiac sympathetic denervation; gene-specific trials.
~1 in 5K. Losartan plus beta-blocker; refined aortic-surveillance and surgical-threshold guidance.
~4M US. Transcatheter edge-to-edge repair expanding; new replacement devices; functional-vs-degenerative pathways.
~10-12M US. Low-dose rivaroxaban plus aspirin (VOYAGER PAD); revascularization and supervised-exercise advances.
~1.6M US with significant TR. Transcatheter tricuspid repair (TriClip, FDA 2024) and replacement systems — first device options.
~900K events/yr US. Catheter-directed and mechanical thrombectomy for selected PE/DVT; refined DOAC duration strategies.
MASLD/MASH (fatty liver disease), fibrosis staging, approved medications (resmetirom, semaglutide), cirrhosis management, liver cancer screening, transplant pathway, and lifestyle interventions.
Available NowUrsodiol, obeticholic acid, elafibranor, fibrate therapy, pruritus management, bone health, transplant evaluation, and monitoring.
Available NowAll ulcer types: peptic ulcers, diabetic foot ulcers, venous leg ulcers, pressure injuries. Diagnosis, treatment, prevention, and patient resources.
~1 in 100K/yr. Per-oral endoscopic myotomy (POEM) widely adopted; high-resolution-manometry subtyping guides therapy.
~1 in 100K (symptomatic rarer). Givosiran (Givlaari) RNAi prophylaxis dramatically reduces attacks; refined diagnostic testing.
~2M US (most undiagnosed). First non-dietary therapies in trials (e.g., gluten-degrading enzymes, immune-tolerance approaches).
~90-150K US. Total pancreatectomy with islet autotransplantation for selected patients; endoscopic and pain-management advances.
~150K+ US, rising fast. Dupilumab (Dupixent) — first FDA-approved drug for EoE; budesonide oral suspension (Eohilia).
~5-6K new/yr US. Ripretinib and avapritinib for specific mutations; mutation-guided sequencing of tyrosine kinase inhibitors.
~5M US with symptoms. Limited drug options; gastric per-oral endoscopic myotomy (G-POEM); newer prokinetic and device research.
~1.9-2.4M US. Understanding your blood markers, antiviral suppression (tenofovir, entecavir), liver-cancer surveillance, perinatal prevention & vaccination, hepatitis D (now treatable with bulevirtide), and the functional-cure pipeline (bepirovirsen).
Available Now~2.4M US. Curable for almost everyone with an 8-12 week course of well-tolerated pills (Mavyret, Epclusa). Universal screening, the 2025 same-visit point-of-care test-and-treat model, cirrhosis care, special populations, drug interactions, and preventing reinfection after cure.
~30-50K US. No approved disease-modifying drug yet; norUDCA, FXR/PPAR agents, and antibiotic-strategy trials active.
~10-20K US on parenteral support. Teduglutide (GLP-2 analog) reduces parenteral-nutrition dependence; next-generation GLP-2 agents (apraglutide).
~9-10K US. New chelation options and trientine formulations; bis-choline tetrathiomolybdate trials; gene therapy programs.
Hydroxyurea, voxelotor, crizanlizumab, L-glutamine, gene therapy (Casgevy, Lyfgenia), pain crisis management, and transition to adult care.
~2-3 per million/yr. Eltrombopag added to immunosuppression as frontline (improves response); transplant-vs-IST decisions refined.
~25-35 new/yr US. Steroids and transfusion standard; gene-therapy and novel-agent trials; ribosomal-genetics insight.
~1 in 130K births. Hematopoietic transplant refinements; gene therapy trials; intensive cancer-surveillance protocols.
~1 in 100K children; adult forms underdiagnosed. Emapalumab (anti-IFN-gamma) approved; refined HLH-2004 and trigger-directed treatment.
Severity classification, factor replacement therapy, non-factor prophylaxis (emicizumab, fitusiran, concizumab, marstacimab), gene therapy (Hemgenix, Roctavian), Altuviiio, inhibitor management, and musculoskeletal care.
Available Now~1 in 50K US. Donidalorsen and garadacimab approved 2025; oral prophylaxis (berotralstat); NTLA-2002 one-time CRISPR therapy in trials.
Available Now~60-125K US. How low platelets are diagnosed, when to treat vs monitor, steroids and IVIG, platelet-boosting TPO agonists, fostamatinib, rituximab and splenectomy, newer agents (rilzabrutinib, FDA-approved 2025), managing bleeding, and pregnancy.
SCID ~1 in 58K births; many IEI types. Gene therapies for SCID and other IEIs; newborn SCID screening now universal in the US.
~15-20K new/yr US. Luspatercept first-line for lower-risk anemia (COMMANDS); imetelstat (Rytelo, 2024); molecular IPSS-M risk model.
Available Now~13-20K US. Momelotinib (2023) for anemic patients; pacritinib; pelabresib and navitoclax combination trials.
~1 in 100K US. Factor B/D inhibitors iptacopan (Fabhalta) and danicopan; pegcetacoplan — oral and proximal-complement options.
~100K US. Ropeginterferon alfa-2b (Besremi) — first interferon approved for PV; rusfertide (hepcidin mimetic) phase 3.
~3-4 per million/yr. Caplacizumab added to plasma exchange and immunosuppression; rituximab; recombinant ADAMTS13 for congenital TTP.
Type 2 remission science, GLP-1 and dual agonist medicines, metabolic surgery, Type 1 technology (CGM, automated insulin delivery), complications, supplements, and action tools.
~64M US with high Lp(a). Pelacarsen, olpasiran, and lepodisiran — Lp(a)-lowering agents in phase 3; first potential targeted therapy.
~1 in 75-200K. Afamelanotide (Scenesse) increases pain-free light exposure; dersimelagon (oral) trials.
~3-5K diagnosed US (underdiagnosed). Pegunigalsidase alfa (Elfabrio); oral chaperone migalastat; gene therapy and substrate-reduction trials.
Available Now Ultra-Rare — First Guide Worldwide~15 patients worldwide. Pseudouridine synthase 7 deficiency causing ID, microcephaly, self-injury. AAV9 gene therapy potential. 4-LLM consensus verified.
~1 in 250 (~1.3M US, mostly undiagnosed). Inclisiran (twice-yearly siRNA), evinacumab for homozygous FH, lerodalcibep; cascade screening.
~6K US. Established enzyme replacement and substrate reduction; gene therapy (PR001/LY3884961) trials; GBA1-Parkinson link research.
~1 in 40-300K. Sebelipase alfa (Kanuma) enzyme replacement — disease-altering for Wolman disease and CESD.
~1 in 5K combined. Supportive 'mito cocktails'; gene-specific and substrate trials; refined diagnostic genomics.
~1 in 100-150K. Levacetylleucine (Aqneursa, 2024) and arimoclomol approved — first disease-targeted options.
~16-20K US. Sepiapterin (Sephience, 2025) — new oral option; pegvaliase (Palynziq) for adults; gene-editing programs.
Available Now~3-5K US. Avalglucosidase alfa (Nexviazyme) and cipaglucosidase alfa + miglustat (Pombiliti) — improved enzyme therapies.
~1 in 15-30K. Diazoxide choline (DCCR) and other trials for hyperphagia; growth-hormone and behavioral management.
~1 in 35K births. Nitrogen-scavenger therapies; liver transplant; mRNA and gene-therapy programs in development.
Curve monitoring, bracing, surgical options (fusion, VBT), physical therapy, pain management, adult degenerative scoliosis, and specialist referral.
Available Now~10K US. Vosoritide (Voxzogo) — first drug to address growth; oral infigratinib (FGFR inhibitor) trials.
~16-40K US. Losmapimod (DUX4-pathway) phase 3 trials; improved genetic diagnostics.
~4M US adults. Refined multimodal management; central-sensitization framework; emerging neuromodulation research.
~9M US (subset refractory). Pegloticase co-administered with methotrexate (improves response/durability); treat-to-target urate lowering.
~2 per 100K. Multiple gene-therapy programs by subtype; precise genetic subtyping increasingly essential.
~1 in 25K combined births. Enzyme replacement; intrathecal and gene-therapy programs (e.g., for Sanfilippo) advancing.
~33M US. No disease-modifying drug yet; trials of NGF inhibitors and structure-modifying agents; weight-loss/GLP-1 impact.
~10M US. Bone-density (DXA) & FRAX fracture risk, calcium/vitamin D & exercise, bisphosphonates and denosumab, bone-building medicines (teriparatide, abaloparatide, romosozumab), anabolic-first sequencing, drug holidays, and the never-stop-denosumab-alone rule.
~700K US. Sarilumab approved for PMR (2023) — first biologic option; steroid-tapering strategies.
~1 in 50-200K. Celiprolol shown to reduce arterial events; genetic confirmation; emergency-planning protocols.
Inhaler therapy optimization, pulmonary rehabilitation, exacerbation prevention, oxygen therapy, lung volume reduction, and end-of-life planning.
Available NowCFTR modulators (Trikafta, Alyftrek), pulmonary management, nutrition, fertility, transplant, and the transition from pediatric to adult care.
Available NowWhat pulmonary fibrosis is and the realistic outlook, getting an accurate diagnosis at an ILD center, antifibrotics explained (nintedanib, pirfenidone, nerandomilast/Jascayd) with side-effect tips, staying active, oxygen, transplant, exacerbation warning signs, family planning & inherited risk, trials, and a hopeful path through every stage.
Available Now~100K symptomatic; vastly underdiagnosed. Augmentation therapy plus emerging RNAi (fazirsiran) for liver disease; gene therapy programs.
Available Now~350-500K US. Brensocatib (DPP-1 inhibitor) — ASPEN trial positive, FDA approval 2025; first disease-modifying drug for bronchiectasis.
~4M+ US. Biologics dupilumab, omalizumab, and mepolizumab — first drug class to shrink polyps and cut surgery.
Hundreds of thousands US (across CTDs). Nintedanib for progressive fibrosing ILD; nerandomilast (FIBRONEER-ILD) expanding antifibrotic options.
Millions US (estimates vary widely). No approved treatment; RECOVER trials and mechanistic studies (immune, autonomic, microclot, viral persistence) maturing.
~3-5 per million women. Sirolimus stabilizes lung function; everolimus and newer mTOR-pathway trials; VEGF-D biomarker.
~150-200K US. How this multisystem granulomatous disease is diagnosed, when to treat vs monitor, steroids and steroid-sparing medicines (methotrexate), infliximab, heart/nerve/eye/skin involvement, managing fatigue, advanced lung disease, and an honest read of the emerging-therapy pipeline.
Available Now~2.5M+ US with severe disease. Tezepelumab (anti-TSLP) covers eosinophil-low patients; expanding biologic landscape; biologic-driven remission as a goal.
~700K US with active disease. Oral JAK inhibitors baricitinib, ritlecitinib, and deuruxolitinib (2024) — first systemic approvals.
~16.5M US adults. Daily skin-barrier care, topical steroids and steroid-free creams, phototherapy, biologic injections (dupilumab plus newer IL-13 and IL-31 options), oral JAK-inhibitor pills, managing itch, sleep and infections, and eczema in children.
~1.5M US at any time. Omalizumab established; dupilumab approved (2025); remibrutinib (BTK inhibitor) trials.
~25-30K US. Beremagene geperpavec (Vyjuvek) — first topical gene therapy (2023); birch-bark gel (Filsuvez); prademagene cell therapy.
~1-7 per million. Spesolimab (Spevigo) — first approved IL-36-receptor antibody for flares and prevention.
~1% of US population. Secukinumab and bimekizumab approved; IL-17 and other biologics expanding beyond adalimumab.
~150-200K US. Dupilumab and nemolizumab approved — first FDA-approved therapies for this intensely itchy disease.
~7.5M US. Bimekizumab (dual IL-17A/F) and deucravacitinib (oral TYK2) — high skin-clearance options.
~2-3M US. Ruxolitinib cream (Opzelura) — first FDA-approved repigmentation therapy; oral JAK inhibitors in trials.
New targeted therapies (sparsentan, iptacopan, budesonide), proteinuria management, dietary considerations, kidney function preservation, and transplant planning.
Available Now~35M US. SGLT2 inhibitors and finerenone slow progression; FLOW trial — semaglutide reduces kidney events; GLP-1 era.
~5-7 per million/yr US. Sparsentan (Filspari) approved with an FSGS indication pathway; targeted trials (e.g., APOL1 inhibitors).
~140K diagnosed US. Genetics and family testing, how fast PKD may progress (Mayo imaging class), blood-pressure control, the kidney-protecting medicine tolvaptan, managing cyst pain/infection, liver cysts, brain-aneurysm screening, and planning for transplant.
~1-3 per million. RNAi therapies lumasiran (Oxlumo) and nedosiran (Rivfloza) — disease-altering for kidney-stone/failure risk.
The first FDA-approved treatments — Syfovre and Izervay — with honest expectations and long-term data, the retinal-vasculitis safety warning, OCT/FAF monitoring, AREDS2, the gene- and cell-therapy pipeline, and low-vision support.
~10M US with some retinopathy. Faricimab and longer-acting anti-VEGF; expanding role of earlier intervention in non-proliferative disease.
~4.2M US. Eye-pressure and optic-nerve testing, pressure-lowering drops (all classes), SLT and Direct SLT laser (now first-line), minimally invasive (MIGS) and traditional surgery, sustained-release implants (iDose TR travoprost, Durysta), and angle-closure emergencies.
~200K US across IRDs. Voretigene (Luxturna) for RPE65 disease; broad gene-therapy and optogenetic pipeline.
~1.5M+ US. Faricimab (dual Ang-2/VEGF) and high-dose aflibercept extend treatment intervals; port delivery system.
~1 in 10 women. How it's diagnosed (often without surgery), pain relief and hormonal treatments, GnRH-antagonist medicines (elagolix, relugolix combination), surgery, fertility and IVF, adenomyosis, and managing chronic pelvic pain.
~5M+ US. GLP-1 receptor agonists increasingly used for metabolic and reproductive features; phenotype-based management.
~1 in 25 US pregnancies. sFlt-1/PlGF ratio biomarker test (FDA-cleared 2023) for short-term risk prediction; aspirin prophylaxis.
~26M US (symptomatic in many). Oral GnRH antagonist combinations (relugolix-Myfembree, elagolix-Oriahnn); uterine-sparing options.
Evidence-based psychotherapy, medication options, treatment-resistant approaches, lifestyle interventions, crisis resources, and when to seek specialist care.
Available NowAntipsychotic selection, clozapine pathway, LAI formulations, cognitive remediation, supported employment, family psychoeducation, and crisis planning.
~1M+ US (lifetime). Higher-calorie refeeding evidence; olanzapine adjunct data; psilocybin and other novel-treatment trials.
A lifelong mood disorder of mania/hypomania and depression. The types (bipolar I, II, cyclothymia), diagnosis, mood stabilizers (lithium, valproate, lamotrigine) and antipsychotics for mania and bipolar depression, staying well and preventing relapse, suicide-safety planning, talk therapy, and pregnancy.
~2.5M US. Refined exposure-based therapy access; deep brain stimulation and TMS for refractory disease.
~6M US. Expanded buprenorphine access; high-dose and extended-release formulations; contingency management.
~13M US in a given year. Trauma-focused therapy evidence; MDMA-assisted therapy under regulatory review; stellate-ganglion-block research.
Insomnia and CBT-I, restless legs syndrome, obstructive sleep apnea, circadian rhythm disorders, sleep studies, medications and devices, and practical implementation.
~600K US. Refined diagnostic criteria; intratympanic therapy; endolymphatic-hydrops imaging research.
~30M US (most undiagnosed). Tirzepatide (SURMOUNT-OSA, FDA Dec 2024) — first drug ever approved for OSA; hypoglossal nerve stimulation expanding.
~66K new/yr US. Time-sensitive corticosteroid therapy (oral and intratympanic); the 'ear emergency' message.
~1.2M US. Long-acting injectable cabotegravir/rilpivirine; lenacapavir (twice-yearly); lenacapavir PrEP (PURPOSE trials, FDA 2025).
~8-9K new/yr US; massive globally. Shorter all-oral regimens (BPaL/BPaLM) for drug-resistant TB; rapid molecular diagnostics.
The most common cause of an overactive thyroid. Diagnosis and antibody (TRAb) tests, antithyroid medicines, radioactive iodine and thyroid surgery, beta-blockers, thyroid eye disease, thyroid-storm warning signs, pregnancy, and life after treatment.
Available Now~15-20K new/yr US. Teprotumumab (Tepezza) — first drug to reduce proptosis without surgery; new IGF-1R agents in development.
A comprehensive guide for hypertrophic cardiomyopathy (HCM): diagnosis, mavacamten (Camzyos), aficamten (Myqorzo), SCD risk and ICD decisions, septal myectomy, exercise guidance, genetic testing, clinical trials, and resources for patients and families.
Available NowA comprehensive guide to Crohn's disease and ulcerative colitis: diagnosis, biologic and small-molecule therapy (IL-23 inhibitors, JAK inhibitors, S1P modulators, anti-TNF, vedolizumab), treat-to-target monitoring, surgery, diet, pregnancy, ASUC management, perianal disease, and practical resources for patients, caregivers, and clinicians.
Available NowA practical, evidence-based guide to beta-thalassemia for patients and caregivers. Covers diagnosis, transfusions, iron chelation, luspatercept, mitapivat, stem cell transplant, and gene therapy (Casgevy and Zynteglo). Updated May 2026.
Available NowPractical guide for patients and families on spinal muscular atrophy (SMA): newborn screening, genetic diagnosis, the three FDA-approved SMN-targeted therapies (Zolgensma, Spinraza, Evrysdi), multidisciplinary care, daily life, and resources including Utah-specific services.
Available NowA practical, evidence-based guide for families and caregivers navigating Duchenne muscular dystrophy: diagnosis and genetic testing, corticosteroid therapy (deflazacort, prednisone, vamorolone with 8-year data), exon-skipping and gene-targeted treatments, the November 2025 Elevidys safety update, givinostat, cardiac and respiratory care, scoliosis and bone health, equipment and school planning, transition to adult life, and the 2025–2026 treatment landscape including next-generation therapies (del-zota, z-rostudirsen, deramiocel, RGX-202, CRISPR gene editing).
Available NowA practical, evidence-based guide for patients and families navigating systemic lupus erythematosus (SLE): diagnosis and blood tests (ANA, anti-dsDNA, complement), hydroxychloroquine as foundation therapy, steroid minimization, biologic therapies (anifrolumab/Saphnelo, belimumab/Benlysta), lupus nephritis treatment (voclosporin/Lupkynis, obinutuzumab/Gazyva approved October 2025), antiphospholipid syndrome, pregnancy planning, health disparities, CAR-T cell therapy for refractory disease, deucravacitinib and emerging therapies, flare management, and living well with lupus.
Available NowA comprehensive, evidence-based guide for endometrial cancer: molecular classification (POLE, MSI-H, p53-abnormal, NSMP), surgery and staging with sentinel lymph node mapping, adjuvant therapy decisions, immunotherapy combinations (dostarlimab, pembrolizumab), targeted therapies (lenvatinib, trastuzumab deruxtecan), fertility preservation, survivorship, and practical resources for patients and families.
Available NowA comprehensive, evidence-based guide for type 1 diabetes: autoantibody screening and staging, insulin therapy optimization, CGM and automated insulin delivery systems, teplizumab immunotherapy, complication prevention, stem cell therapies, pregnancy management, psychosocial care, and practical resources for patients and families.
Available NowA comprehensive guide for balance and dizziness disorders: BPPV, Meniere's disease, vestibular migraine, vestibular neuritis, PPPD, vestibular rehabilitation, falls prevention, and practical resources for patients and families.
Available NowA comprehensive guide for AL amyloidosis (light chain amyloidosis): diagnosis, amyloid typing, treatment options including daratumumab, stem cell transplant, clinical trials, supportive care, and resources for patients and families.
Available NowA comprehensive guide to transient global amnesia (TGA), transient epileptic amnesia (TEA), transient ischemic attack (TIA), and related conditions — for patients, families, and healthcare professionals.
Available NowA comprehensive guide to autism spectrum disorder (ASD): diagnosis, evidence-based therapies, medications for co-occurring conditions, educational rights, transition to adulthood, and support resources for individuals and families.
Available NowA comprehensive guide to PCOS and early menopause (POI): diagnosis, treatment, fertility options, metabolic health, hormone replacement, and support resources for women and families.
Available NowA comprehensive guide to ADHD and OCD: diagnosis, medications, therapy (CBT/ERP), managing co-occurring conditions, school and workplace accommodations, daily strategies, and support resources for patients and families.
Available NowA comprehensive guide to asthma: diagnosis, inhaler therapy, ICS-formoterol reliever strategy, asthma action plans, trigger management, severe asthma biologics, and practical daily management for patients and families of all ages.
Evidence-based clinical decision support for healthcare practitioners — structured data tables, treatment algorithms, regimen dosing, guideline concordance, and adverse event management.
TNM staging, molecular profiling, adjuvant regimen dosing (IDEA framework), biomarker-directed metastatic therapy, AE management, and NCCN surveillance.
View → Available Now Clinical AlgorithmsWHO 2021 classification, MGMT-guided therapy, Stupp protocol, TTFields (EF-14), elderly adaptations, RANO response criteria, and dexamethasone management.
View → Available Now Clinical AlgorithmsBiomarker-directed treatment algorithms, perioperative strategies, immunotherapy, targeted therapy sequencing, resistance management, and decision support.
View → Available Now Clinical AlgorithmsIMWG diagnostic criteria, R-ISS risk stratification, quadruplet frontline therapy, ASCT, bispecific antibodies, CAR-T, MRD-guided approaches, and treatment algorithms.
View → Available Now Clinical AlgorithmsWHO 2022 classification, Lugano staging, R-CHOP/Pola-R-CHP for DLBCL, follicular lymphoma, mantle cell protocols, CAR-T and bispecific antibodies, prognostic indices.
View → Available Now Clinical AlgorithmsResectability & molecular profiling, neoadjuvant strategy, surgical/volume principles, PRODIGE-24 adjuvant & first-line selection (incl. NALIRIFOX/NAPOLI-3), the KRAS frontier (daraxonrasib/RASolute 302), PARP maintenance, PERT/biliary/VTE/pain management, and evidence-graded tables.
View → Available Now Clinical AlgorithmsNCCN risk stratification, ISUP grading, active surveillance protocols, radiation dose-escalation, mCSPC intensification trials, mCRPC sequencing, and PSMA-targeted therapy.
View → Available Now Clinical AlgorithmsSubtype-directed algorithms (HR+/HER2-, HER2+, HER2-low, TNBC), biomarker panel and IHC cutoffs, neoadjuvant/adjuvant protocols, metastatic sequencing, ADC and CDK4/6 toxicity management, dosing tables, and evidence grades.
View → Available Now Clinical AlgorithmsATA 2015/2023 guidelines, NCCN v2.2026, molecular-guided FNA, Bethesda-to-surgery algorithms, RAI dosimetry, dynamic risk stratification, RET/BRAF/NTRK-directed therapy, and MTC monitoring.
View → Available Now~6K new/yr US. Blinatumomab moving into frontline consolidation (E1910); CAR-T (brexucabtagene, tisagenlecleucel); MRD-guided therapy.
Available Now~20K new/yr; ~11K deaths. Menin inhibitor revumenib (Revuforj, FDA 2024) for KMT2A/NPM1; expanding IDH1/2 and FLT3 inhibitors; oral azacitidine maintenance.
~1-2 per million/yr. Mitotane plus chemotherapy standard; immunotherapy and targeted trials; hormone-excess management.
~10K new/yr US. Immunotherapy in advanced disease; HPV-driven prevention and high-resolution-anoscopy screening (ANCHOR).
~83K new/yr US. Enfortumab vedotin + pembrolizumab (EV-302) transformed first-line metastatic; nadofaragene and N-803 for BCG-unresponsive NMIBC.
~5-6K new/yr US (all types). Siltuximab for idiopathic multicentric disease; sirolimus and JAK-pathway trials; CDCN-driven research.
~14K new/yr US. Pembrolizumab + chemoradiation (KEYNOTE-A18); tisotumab vedotin for recurrent disease; HPV-driven prevention.
Available Now~8-12K new/yr US. FGFR2 inhibitors (pemigatinib, futibatinib), IDH1 inhibitor ivosidenib, durvalumab+chemo (TOPAZ-1) first-line.
Available Now~21K new/yr; ~200K prevalence. Fixed-duration BTK+BCL2 (venetoclax-based) combinations; pirtobrutinib for BTK-resistant disease.
~3K new/yr US. Mogamulizumab; expanding skin-directed and systemic options; stage-driven therapy.
~5-6 per million/yr. Nirogacestat (Ogsiveo, 2023) — first FDA-approved therapy; gamma-secretase inhibition.
~25K new/yr US. Bispecifics epcoritamab and glofitamab; pola-R-CHP first-line (POLARIX); CAR-T moved to second line.
~300-400 new/yr US. Dordaviprone (Modeyso) accelerated approval (2025) for H3 K27M-mutant disease — first targeted option.
~67K new/yr. Dostarlimab+chemo (RUBY) and pembrolizumab+chemo (KEYNOTE-868/NRG-GY018) reshaped first-line advanced/recurrent disease; molecular classification.
Staging discipline, mandatory biomarkers (PD-L1/HER2/MMR/CLDN18.2), ESOPEC (FLOT>CROSS for adeno), CheckMate-577 adjuvant nivolumab, first-line IO sequencing, HER2/T-DXd, irAE management, and evidence-graded tables throughout.
~200 new/yr US. Refined multimodal therapy; trials of targeted agents; survivorship-focused care.
~27K new/yr US. Zolbetuximab (Vyloy) for Claudin 18.2-positive tumors; trastuzumab deruxtecan; IO by PD-L1 status.
Clinical AlgorithmsSubsite & HPV/EBV-stratified staging, cisplatin chemoradiation, perioperative pembrolizumab (KEYNOTE-689), CPS-guided R/M immunotherapy, nasopharyngeal carcinoma, organ preservation & toxicity — evidence tables.
View → Available Now~42K new/yr US. Atezolizumab+bevacizumab and durvalumab+tremelimumab first-line; IMbrave050 adjuvant data; expanding earlier-stage IO.
~5-6K new/yr US. Vorasidenib (Voranigo, FDA 2024) — first targeted therapy for grade 2 IDH-mutant glioma (INDIGO trial).
~100K new/yr US. Tumor-infiltrating lymphocyte therapy lifileucel (Amtagvi, 2024); adjuvant IO; mRNA-4157 personalized vaccine (with pembrolizumab) phase 3.
~3K new/yr US. Avelumab and pembrolizumab — checkpoint inhibitors transformed this aggressive skin cancer; neoadjuvant IO trials.
~3K new/yr US. Nivolumab+ipilimumab first-line (CheckMate-743); perioperative IO trials; ADCs in development.
~700-800 new/yr US. Anti-GD2 immunotherapy (dinutuximab, naxitamab) standard in high-risk disease; eflornithine maintenance.
~1K new/yr US. Limb-salvage surgery advances; immunotherapy and targeted trials; metastatic-disease research.
~20K new/yr; ~12K deaths. PARP-inhibitor maintenance (HRD-guided); mirvetuximab soravtansine for folate-receptor-alpha-positive disease.
Available Now~82K new/yr US. IO-TKI combinations standard; belzutifan (HIF-2alpha inhibitor) for VHL and advanced disease; adjuvant pembrolizumab.
~200-300 new/yr US. Intra-arterial and intravitreal chemotherapy enable eye preservation; genetic (RB1) testing and counseling.
VALG/TNM staging, limited-stage chemoradiation + consolidation durvalumab, extensive-stage chemoimmunotherapy (IMpower133/CASPIAN/IMforte), DLL3-targeted tarlatamab with CRS/ICANS management, regimen dosing, CNS & paraneoplastic care, and the molecular-subtype pipeline.
View →~13K new/yr US. Subtype-specific advances; nirogacestat for desmoids; afamitresgene autoleucel (T-cell therapy) for synovial sarcoma (2024).
~400-500 new/yr US. Refined multimodal therapy; immunotherapy use tempered by autoimmune risk; paraneoplastic awareness.
~2.5K new/yr US. Tebentafusp (Kimmtrak) — first therapy to extend overall survival in metastatic disease (HLA-A*02:01 patients).
NIA-AA revised diagnostic criteria, plasma p-tau217 biomarker integration, anti-amyloid antibody protocols, ARIA monitoring, APOE-stratified risk, and treatment algorithms.
View → Available Now Clinical AlgorithmsRevised El Escorial and Gold Coast criteria, ALSFRS-R/King’s staging, genetic evaluation, pharmacotherapy, NIV indications, gastrostomy timing, symptomatic management, and palliative care.
View → Available Now Clinical AlgorithmsMDS 2022 MSA criteria, autonomic management, supplement evidence grading, and clinical decision support for alpha-synucleinopathies.
View → Available Now Clinical AlgorithmsAntibody-driven classification, MGFA staging, FcRn inhibitor selection, complement inhibitor management, thymectomy, crisis protocols, and treatment algorithms.
View → Available Now Clinical AlgorithmsMDS diagnostic criteria, levodopa formulations, dopamine agonist dosing, COMT/MAO-B inhibitors, DBS patient selection, motor fluctuation algorithms, and non-motor management.
View →~1 in 12-20K. Antisense-oligonucleotide programs targeting UBE3A in trials; symptomatic-care optimization.
~1 in 25-50K. Genetic diagnosis (NOTCH3); supportive management; emerging therapeutic research.
~1 in 200-600. Propranolol and other repurposed-drug trials; familial-form genetic testing; imaging-surveillance guidance.
Demyelinating-vs-axonal classification and electrodiagnosis, genetic-testing pathway, the CMT-vs-CIDP/ATTR decision pathway, neurotoxic-drug stewardship (vincristine contraindicated), anesthesia, management with evidence grades, and the gene-directed pipeline.
View →~20-30K US. Efgartigimod (Vyvgart) approved for CIDP (2024); maintenance immunoglobulin optimization.
~4M US with chronic migraine. CGRP monoclonal antibodies and oral gepants for prevention and acute use; growing real-world evidence.
~1 in 1K. Galcanezumab for episodic cluster; non-invasive vagus nerve stimulation; oxygen and acute-treatment optimization.
~1 in 15-16K births. Fenfluramine and cannabidiol approved; STK-001 antisense oligonucleotide and gene-therapy trials.
~1M US with refractory seizures. New antiseizure medications (cenobamate); responsive neurostimulation and laser ablation; surgical-evaluation push.
Two-axis classification, the treatable-mimic workup (levodopa trial, Wilson screen), botulinum toxin selection/dosing (incl. daxibotulinumtoxinA), oral agents, GPi-DBS (now full FDA PMA 2025), status dystonicus, acute dystonic reactions, combined/paroxysmal forms, and the non-motor/psychosocial burden.
View →~7M US. MRI-guided focused ultrasound thalamotomy — incisionless option; expanding bilateral treatment.
~1 in 4-7K. Targeted trials (e.g., zatolmilast/BPN14770 PDE4D inhibitor) for cognition; refined behavioral care.
~5K US. Omaveloxolone (Skyclarys, 2023) — first-ever approved treatment; gene therapy and frataxin-replacement programs advancing.
Available Now~50-60K US. Progranulin-raising therapies (latozinemab) and C9orf72-targeted ASOs in trials; AC Immune and others advancing.
~1-2 per 100K/yr. IVIG and plasma exchange standard; complement inhibitor (eculizumab) and other trials for severe disease.
~10-40K US (underdiagnosed). TTR silencers patisiran, vutrisiran, eplontersen; NTLA-2001 (Nextriva) one-time CRISPR therapy advancing.
~1-5 per 100K. Gene-discovery expansion; subtype-specific trials emerging; supportive-care optimization.
~41K symptomatic US; ~200K at-risk. AMT-130 AAV gene therapy showed first-ever signal of slowed progression (2025); huntingtin-lowering ASO programs continuing.
~3 per million. Amifampridine (Firdapse/Ruzurgi); strong association with small-cell lung cancer driving cancer screening.
~100 new/yr US. Idebenone; lenadogene nolparvovec gene-therapy trials; mitochondrial-targeted approaches.
~30-50K US. Cannabidiol and fenfluramine approved; cenobamate and other agents studied; dietary and device options.
~1.4M US. Refined biomarkers (skin/CSF alpha-synuclein seed amplification); neuroleptic-sensitivity awareness; symptomatic-care advances.
Available NowVasCog-2-WSO (2025) criteria and subtypes, imaging workup, vascular risk-factor control and stroke prevention (SPRINT-MIND; AF anticoagulation vs microbleed/CAA risk), symptomatic cognitive/mood pharmacotherapy, mixed and monogenic (CADASIL) disease, and referral — for clinicians.
Available NowThe most common substrate of dementia in older adults (Alzheimer’s + cerebrovascular). Treat both processes; anti-amyloid eligibility tempered by vascular/microbleed burden.
Available NowDiagnostic criteria and imaging (Evans index, DESH, callosal angle), tap test and extended lumbar drainage, shunt selection/technique and valve titration, the placebo-controlled PENS RCT (gait benefit; cognition/bladder less reliable), comorbid neurodegeneration, complications, and referral — for clinicians.
Available NowMDS criteria and the 1-year rule (PDD vs DLB), cognitive profile, cholinesterase-inhibitor and neuropsychiatric algorithms, the neuroleptic-sensitivity protocol, deprescribing, delirium workup, hospital safety, and the investigational pipeline (blarcamesine, neflamapimod; ambroxol negative).
Available NowPathophysiology, Caine-criteria clinical diagnosis, empiric parenteral high-dose thiamine before glucose (treat on suspicion), dosing evidence (Dingwall RCT), the under-recognition problem, non-alcoholic WE, Korsakoff prognosis/rehabilitation, and prevention protocols (2025 ASMBS) — for clinicians.
Available NowFrascati ANI/MND/HAD with the 2023 consensus refinement, neuropsychological workup, rigorous confounder exclusion, CSF viral escape, the CPE debate, aging/vascular comorbidity, adherence and long-acting ART, and supportive care — for clinicians.
Available NowRapidly progressive prion dementia with myoclonus and ataxia; RT-QuIC + DWI-MRI diagnosis. No disease-modifying treatment — supportive/palliative; median survival months.
~1-2 per 100K. Newly defined entity (MOGAD); rozanolixizumab and other trials; distinguished from MS and NMOSD.
~1 in 100K US (higher in East Asia). Revascularization surgery (bypass) is mainstay; refined patient selection and imaging.
DM1/DM2 genetics & spliceopathy, cardiac conduction surveillance & device decisions (sudden-death prevention), the anesthetic-risk protocol, mexiletine for myotonia, congenital DM1, pregnancy, multisystem management with evidence grades, and the RNA-targeted pipeline (del-desiran).
View →~150-200K US. Orexin-receptor agonists (oveporexton/TAK-861) in phase 3 — potentially disease-targeted, not just symptomatic.
~12K new/yr US; rising prevalence. Lutetium-177 dotatate (Lutathera) expanding to earlier lines; somatostatin-analog and targeted-agent advances.
2021 diagnostic criteria (vs Legius/mosaic), age-stratified surveillance, the MPNST/ANNUBP malignant-transformation algorithm, optic pathway glioma, MEK-inhibitor therapy & monitoring, the cognitive phenotype, treatable vascular/endocrine workup, breast-cancer surveillance, transition of care, and the pipeline.
View → Available Now~15K US. Four approved biologics: eculizumab, satralizumab, inebilizumab, and ravulizumab (2024) — relapse prevention transformed.
~7-10M US with significant symptoms. Shift away from dopamine agonists (augmentation risk) toward alpha-2-delta ligands and iron repletion.
~6-9K US. Trofinetide (Daybue, 2023) — first-ever approved drug; gene-therapy programs (e.g., TSHA-102) in trials.
~10-25K US. Onasemnogene (Zolgensma) gene therapy, nusinersen (Spinraza), risdiplam (Evrysdi); newer higher-dose and combination strategies.
~1-5 per 100K. Troriluzole and gene-specific antisense programs in trials; expanded genetic testing.
~150K US. Microvascular decompression and modern radiosurgery/ablation; newer sodium-channel-blocker research.
~50K US. mTOR inhibitors (everolimus) for SEGAs/angiomyolipomas; cannabidiol for seizures; surveillance protocols.
2024 McDonald criteria, DMT algorithms, progressive MS treatment including BTK inhibitors, safety monitoring, and decision frameworks for healthcare practitioners.
View → Available Now~40-50K US. Avacopan (Tavneos) — oral C5a-receptor blocker enabling major steroid reduction; rituximab maintenance optimization.
~1-2 per million/yr. IL-1 (anakinra, canakinumab) and IL-6 inhibitors; recognition of macrophage-activation-syndrome risk.
~100-200K US. Standard steroid/azathioprine plus emerging steroid-sparing strategies; refined diagnostic scoring.
~1.5-3M US (incl. non-radiographic). IL-17 inhibitors and JAK inhibitors; non-radiographic axSpA recognition; earlier diagnosis push.
~1 in 20K US (higher along Silk Road). Apremilast for oral ulcers; TNF and IL-1 inhibitors; organ-specific treatment frameworks.
~2-5 per 100K/yr (elderly). Dupilumab approved for bullous pemphigoid (2025) — first targeted biologic; steroid-sparing.
~10 per 100K. IVIG (Octagam 10%) approved for dermatomyositis; myositis-specific antibody-driven subtyping; JAK inhibitors.
~1 in 200-1K in at-risk populations. Colchicine remains first-line; IL-1 inhibitors for colchicine-resistant disease; MEFV genetic testing.
~228K US over 50. Tocilizumab steroid-sparing; new IL-6 and GM-CSF-pathway trials; fast-track diagnostic pathways.
~1-5 per 100K (likely underdiagnosed). Inebilizumab (MITIGATE trial, 2025) — first agent with a dedicated IgG4-RD program; rituximab widely used.
~5-10 per 100K over 50. No approved treatment; ulnar/finger-flexor pattern recognition; trials of myostatin and autophagy-targeted agents.
~1-5 per million/yr. Rituximab first-line (with steroids) is now standard; FcRn and other targeted agents in trials.
~1.5M US. IL-17, IL-23, and JAK inhibitors; bimekizumab; domain-based treatment selection.
ACR/EULAR classification & disease-activity assessment, treat-to-target, conventional/biologic/targeted synthetic DMARDs, JAK-inhibitor safety (ORAL Surveillance), extra-articular disease & pregnancy — evidence tables.
View → Clinical Algorithms~0.4-3.1M US (wide estimates). Ianalumab, dazodalibep, and other B-cell/co-stimulation agents in phase 3 — first potential disease-modifying drugs.
View →~1-2 per million. No approved disease-specific drug; rituximab and immunotherapy used off-label; GAD/glycine-receptor antibody subtyping.
~200K-300K US SLE. Anifrolumab uptake, deucravacitinib trials, and dramatic CD19 CAR-T results (Erlangen) producing drug-free remission.
~100K US. Tocilizumab and nintedanib for SSc-ILD; autologous stem-cell transplant; CD19 CAR-T trials showing remission.
CHA2DS2-VASc-guided anticoagulation, PFA vs thermal ablation, early rhythm control, LAAO selection, antiarrhythmic protocols, and special population algorithms.
View → Available Now Clinical AlgorithmsNon-invasive diagnostic algorithm, TTR stabilizer comparison, gene silencer protocols, ATTR-specific HF management, and ACC-aligned decision pathways.
View → Available Now Clinical AlgorithmsGDMT four-pillar optimization, HFpEF therapeutics, ATTR-CM diagnosis and treatment, device therapy algorithms, advanced HF management, and guideline-aligned protocols.
View → Available Now Clinical AlgorithmsHemodynamic diagnosis, ESC/ERS risk-stratified algorithms, combination therapy sequencing, sotatercept integration, transplant referral criteria, and monitoring protocols.
View → Available Now Clinical AlgorithmsIV alteplase/tenecteplase protocols, mechanical thrombectomy selection (DAWN/DEFUSE 3), ICH management, SAH grading, anticoagulant reversal, and secondary prevention.
View →~1.5M+ US (moderate-severe). EARLY TAVR (2024) supports earlier intervention in asymptomatic severe disease; TAVR durability data maturing.
~1 in 2-5K. Genetic-testing-driven diagnosis; exercise-restriction evidence; risk-stratified ICD decisions.
~1 in 2K (higher in Asia). Risk-stratification refinements; ajmaline-challenge use; ICD and ablation decision frameworks.
Underdiagnosed subset of sarcoidosis. Advanced imaging (cardiac MRI/FDG-PET) recognition; immunosuppression plus device therapy protocols.
LDL-C targets & the full lipid-lowering toolkit (PCSK9 mAbs, inclisiran, bempedoic acid, lerodalcibep, investigational oral PCSK9 & Lp(a) agents), residual inflammatory & Lp(a) risk, antithrombotic strategy, SGLT2i/GLP-1 cardiometabolic therapy, revascularization, and a dosing/monitoring appendix — evidence-graded tables & guideline concordance.
View →~1 in 5K (largely undiagnosed). Pomalidomide (PATH-HHT trial, 2024) reduced bleeding; anti-VEGF strategies; systemic therapy emerging.
~700K US (most undiagnosed). Cardiac myosin inhibitors: mavacamten (Camzyos) and aficamten (SEQUOIA-HCM, FDA filing) — first disease-specific drug class.
~1 in 2K. Genotype-specific management; beta-blocker selection by subtype; left cardiac sympathetic denervation; gene-specific trials.
~1 in 5K. Losartan plus beta-blocker; refined aortic-surveillance and surgical-threshold guidance.
~4M US. Transcatheter edge-to-edge repair expanding; new replacement devices; functional-vs-degenerative pathways.
~10-12M US. Low-dose rivaroxaban plus aspirin (VOYAGER PAD); revascularization and supervised-exercise advances.
~1.6M US with significant TR. Transcatheter tricuspid repair (TriClip, FDA 2024) and replacement systems — first device options.
~900K events/yr US. Catheter-directed and mechanical thrombectomy for selected PE/DVT; refined DOAC duration strategies.
MASLD/MASH nomenclature, non-invasive fibrosis staging (FIB-4/VCTE pathway), approved pharmacotherapy, cirrhosis management, HCC surveillance, and transplant referral criteria.
View → Available Now Clinical AlgorithmsDiagnostic workup, UDCA response criteria, PPAR agonist therapy, pruritus management ladder, PBC-AIH overlap, transplant evaluation, and AASLD/EASL-aligned pathways.
View → Available Now Clinical AlgorithmsH. pylori eradication, GI bleeding protocols, wound care algorithms, compression therapy, offloading, and pressure injury prevention.
View →~1 in 100K/yr. Per-oral endoscopic myotomy (POEM) widely adopted; high-resolution-manometry subtyping guides therapy.
~1 in 100K (symptomatic rarer). Givosiran (Givlaari) RNAi prophylaxis dramatically reduces attacks; refined diagnostic testing.
~2M US (most undiagnosed). First non-dietary therapies in trials (e.g., gluten-degrading enzymes, immune-tolerance approaches).
~90-150K US. Total pancreatectomy with islet autotransplantation for selected patients; endoscopic and pain-management advances.
~150K+ US, rising fast. Dupilumab (Dupixent) — first FDA-approved drug for EoE; budesonide oral suspension (Eohilia).
~5-6K new/yr US. Ripretinib and avapritinib for specific mutations; mutation-guided sequencing of tyrosine kinase inhibitors.
~5M US with symptoms. Limited drug options; gastric per-oral endoscopic myotomy (G-POEM); newer prokinetic and device research.
Phase classification, AASLD/IDSA 2025 treatment indications, nucleos(t)ide/peginterferon selection, HCC surveillance, PMTCT, reactivation prophylaxis, hepatitis D (bulevirtide), and the functional-cure pipeline.
View → Available Now Clinical AlgorithmsUniversal screening & non-invasive fibrosis staging, pangenotypic DAA regimen selection, the AASLD/IDSA simplified & 2025 point-of-care test-and-treat algorithms, drug-interaction & regimen-selection tables, special populations (cirrhosis, HIV, CKD/dialysis, pregnancy, PWID, transplant), and post-SVR surveillance — evidence-graded.
View →~3.1M US. Risankizumab, mirikizumab, upadacitinib, etrasimod, guselkumab — multiple new mechanisms 2023-2025; treat-to-target with intestinal ultrasound.
~30-50K US. No approved disease-modifying drug yet; norUDCA, FXR/PPAR agents, and antibiotic-strategy trials active.
~10-20K US on parenteral support. Teduglutide (GLP-2 analog) reduces parenteral-nutrition dependence; next-generation GLP-2 agents (apraglutide).
~9-10K US. New chelation options and trientine formulations; bis-choline tetrathiomolybdate trials; gene therapy programs.
Genotype-specific care, VOC protocols, disease-modifying therapy optimization, gene therapy and transplant patient selection, complication surveillance, and guidelines.
View →~2-3 per million/yr. Eltrombopag added to immunosuppression as frontline (improves response); transplant-vs-IST decisions refined.
~1K+ transfusion-dependent US; far larger globally. Exa-cel (Casgevy) CRISPR cure; betibeglogene (Zynteglo) gene therapy; luspatercept and mitapivat reduce transfusion burden.
~25-35 new/yr US. Steroids and transfusion standard; gene-therapy and novel-agent trials; ribosomal-genetics insight.
~1 in 130K births. Hematopoietic transplant refinements; gene therapy trials; intensive cancer-surveillance protocols.
~1 in 100K children; adult forms underdiagnosed. Emapalumab (anti-IFN-gamma) approved; refined HLH-2004 and trigger-directed treatment.
WFH/ISTH/NHF/ASH guidelines, severity-driven prophylaxis, PK-guided dosing, non-factor therapy positioning, gene therapy eligibility, inhibitor management, and perioperative protocols.
View → Available Now~1 in 50K US. Donidalorsen and garadacimab approved 2025; oral prophylaxis (berotralstat); NTLA-2002 one-time CRISPR therapy in trials.
Clinical AlgorithmsDiagnosis of exclusion & bleeding-risk assessment, first-line corticosteroids & IVIG, second-line TPO-RAs, fostamatinib, rituximab & splenectomy, emerging BTK (rilzabrutinib)/FcRn (efgartigimod) agents, bleeding emergencies & pregnancy — evidence tables.
View →SCID ~1 in 58K births; many IEI types. Gene therapies for SCID and other IEIs; newborn SCID screening now universal in the US.
~15-20K new/yr US. Luspatercept first-line for lower-risk anemia (COMMANDS); imetelstat (Rytelo, 2024); molecular IPSS-M risk model.
Available Now~13-20K US. Momelotinib (2023) for anemic patients; pacritinib; pelabresib and navitoclax combination trials.
~1 in 100K US. Factor B/D inhibitors iptacopan (Fabhalta) and danicopan; pegcetacoplan — oral and proximal-complement options.
~100K US. Ropeginterferon alfa-2b (Besremi) — first interferon approved for PV; rusfertide (hepcidin mimetic) phase 3.
~3-4 per million/yr. Caplacizumab added to plasma exchange and immunosuppression; rituximab; recombinant ADAMTS13 for congenital TTP.
ADA/EASD consensus algorithm, SGLT2i and GLP-1 RA outcome data, insulin titration protocols, complication screening, DKA/HHS management, and individualized A1C targets.
View →~4-5K new/yr US. Daratumumab-based regimens (ANDROMEDA) now standard; CAEL-101 and birtamimab fibril-directed trials.
~64M US with high Lp(a). Pelacarsen, olpasiran, and lepodisiran — Lp(a)-lowering agents in phase 3; first potential targeted therapy.
~1 in 75-200K. Afamelanotide (Scenesse) increases pain-free light exposure; dersimelagon (oral) trials.
~3-5K diagnosed US (underdiagnosed). Pegunigalsidase alfa (Elfabrio); oral chaperone migalastat; gene therapy and substrate-reduction trials.
Available Now Ultra-Rare — First Guide Worldwide~15 patients worldwide. Pseudouridine synthase 7 deficiency. Allelic variants, diagnostic workup, gene therapy feasibility, epitranscriptomic comparison.
~1 in 250 (~1.3M US, mostly undiagnosed). Inclisiran (twice-yearly siRNA), evinacumab for homozygous FH, lerodalcibep; cascade screening.
~6K US. Established enzyme replacement and substrate reduction; gene therapy (PR001/LY3884961) trials; GBA1-Parkinson link research.
~1 in 40-300K. Sebelipase alfa (Kanuma) enzyme replacement — disease-altering for Wolman disease and CESD.
~1 in 5K combined. Supportive 'mito cocktails'; gene-specific and substrate trials; refined diagnostic genomics.
~1 in 100-150K. Levacetylleucine (Aqneursa, 2024) and arimoclomol approved — first disease-targeted options.
~16-20K US. Sepiapterin (Sephience, 2025) — new oral option; pegvaliase (Palynziq) for adults; gene-editing programs.
Available Now~3-5K US. Avalglucosidase alfa (Nexviazyme) and cipaglucosidase alfa + miglustat (Pombiliti) — improved enzyme therapies.
~1 in 15-30K. Diazoxide choline (DCCR) and other trials for hyperphagia; growth-hormone and behavioral management.
~1.6-2M US. Teplizumab (Tzield) delays stage-3 onset; donislecel (Lantidra) allogeneic islet cells; VX-880/zimislecel stem-cell trials; automated insulin delivery...
~1 in 35K births. Nitrogen-scavenger therapies; liver transplant; mRNA and gene-therapy programs in development.
Cobb angle assessment, Lenke and SRS-Schwab classification, bracing protocols, scoliosis-specific exercise evidence, fusion, VBT, and SRS/SOSORT-aligned pathways.
View → Available Now~10K US. Vosoritide (Voxzogo) — first drug to address growth; oral infigratinib (FGFR inhibitor) trials.
~12-15K US. Delandistrogene moxeparvovec (Elevidys) gene therapy — label expanded 2024; exon-skipping agents; givinostat (first non-steroid for all genotypes).
~16-40K US. Losmapimod (DUX4-pathway) phase 3 trials; improved genetic diagnostics.
~4M US adults. Refined multimodal management; central-sensitization framework; emerging neuromodulation research.
~9M US (subset refractory). Pegloticase co-administered with methotrexate (improves response/durability); treat-to-target urate lowering.
~2 per 100K. Multiple gene-therapy programs by subtype; precise genetic subtyping increasingly essential.
~1 in 25K combined births. Enzyme replacement; intrathecal and gene-therapy programs (e.g., for Sanfilippo) advancing.
~33M US. No disease-modifying drug yet; trials of NGF inhibitors and structure-modifying agents; weight-loss/GLP-1 impact.
DXA/FRAX fracture-risk stratification, antiresorptive & anabolic pharmacotherapy, anabolic-first sequencing, drug holidays & the denosumab-discontinuation protocol, glucocorticoid-induced & male osteoporosis, secondary causes — evidence tables.
View →~700K US. Sarilumab approved for PMR (2023) — first biologic option; steroid-tapering strategies.
~1 in 50-200K. Celiprolol shown to reduce arterial events; genetic confirmation; emergency-planning protocols.
GOLD 2025/2026 algorithms, spirometric staging, eosinophil-guided ICS decisions, dupilumab and mepolizumab for type 2 inflammation, ensifentrine, and treatment pathways.
View → Available Now Clinical AlgorithmsNewborn screening algorithms, CFTR genotype-modulator eligibility, Alyftrek vs Trikafta sequencing, pulmonary management, transplant referral, and emerging therapies.
View → Available Now Clinical AlgorithmsHRCT/MDD diagnosis, antifibrotic selection & sequencing (incl. nerandomilast/FIBRONEER, the first add-on for confirmed progression), PPF operationalized, AE-IPF management & goals of care, comorbidity & drug-interaction pharmacology, transplant timing, and guideline-aligned evidence tables.
View → Available Now~100K symptomatic; vastly underdiagnosed. Augmentation therapy plus emerging RNAi (fazirsiran) for liver disease; gene therapy programs.
Available Now~350-500K US. Brensocatib (DPP-1 inhibitor) — ASPEN trial positive, FDA approval 2025; first disease-modifying drug for bronchiectasis.
~4M+ US. Biologics dupilumab, omalizumab, and mepolizumab — first drug class to shrink polyps and cut surgery.
Hundreds of thousands US (across CTDs). Nintedanib for progressive fibrosing ILD; nerandomilast (FIBRONEER-ILD) expanding antifibrotic options.
Millions US (estimates vary widely). No approved treatment; RECOVER trials and mechanistic studies (immune, autonomic, microclot, viral persistence) maturing.
~3-5 per million women. Sirolimus stabilizes lung function; everolimus and newer mTOR-pathway trials; VEGF-D biomarker.
Diagnosis & exclusion of mimics, Scadding staging, treatment indications, glucocorticoid/steroid-sparing/anti-TNF ladder (PREDMETH), cardiac/neuro/ocular disease, fibrotic disease & pulmonary hypertension, and an evidence-based read of the pipeline (EFZO-FIT, RESOLVE-Lung) — evidence tables.
View → Available Now~2.5M+ US with severe disease. Tezepelumab (anti-TSLP) covers eosinophil-low patients; expanding biologic landscape; biologic-driven remission as a goal.
~700K US with active disease. Oral JAK inhibitors baricitinib, ritlecitinib, and deuruxolitinib (2024) — first systemic approvals.
Diagnosis & severity assessment, barrier/topical therapy & phototherapy, type-2 biologics (dupilumab, tralokinumab, lebrikizumab, nemolizumab), oral JAK inhibitors & boxed-warning safety, conventional systemics, the atopic march, and lifespan management — evidence tables.
View →~1.5M US at any time. Omalizumab established; dupilumab approved (2025); remibrutinib (BTK inhibitor) trials.
~25-30K US. Beremagene geperpavec (Vyjuvek) — first topical gene therapy (2023); birch-bark gel (Filsuvez); prademagene cell therapy.
~1-7 per million. Spesolimab (Spevigo) — first approved IL-36-receptor antibody for flares and prevention.
~1% of US population. Secukinumab and bimekizumab approved; IL-17 and other biologics expanding beyond adalimumab.
~150-200K US. Dupilumab and nemolizumab approved — first FDA-approved therapies for this intensely itchy disease.
~7.5M US. Bimekizumab (dual IL-17A/F) and deucravacitinib (oral TYK2) — high skin-clearance options.
~2-3M US. Ruxolitinib cream (Opzelura) — first FDA-approved repigmentation therapy; oral JAK inhibitors in trials.
Targeted therapies (sparsentan, iptacopan, budesonide), proteinuria management, KDIGO-aligned treatment algorithms, and transplant considerations.
View → Available Now~35M US. SGLT2 inhibitors and finerenone slow progression; FLOW trial — semaglutide reduces kidney events; GLP-1 era.
~5-7 per million/yr US. Sparsentan (Filspari) approved with an FSGS indication pathway; targeted trials (e.g., APOL1 inhibitors).
Genetics & diagnosis, total-kidney-volume/genotype progression risk (Mayo class, PROPKD), RAAS-based BP control, tolvaptan therapy & REMS monitoring, cyst-complication & polycystic-liver management, intracranial-aneurysm screening, and kidney replacement — aligned to KDIGO 2025, with evidence tables.
View →~1-3 per million. RNAi therapies lumasiran (Oxlumo) and nedosiran (Rivfloza) — disease-altering for kidney-stone/failure risk.
C3 vs C5 inhibitor selection (Syfovre/Izervay), evidence tables and long-term extension data, cRORA/iRORA imaging, the Dec-2024 retinal-vasculitis warning and AE workup, the function-endpoint pipeline (vonaprument, gene/cell therapy), and decision frameworks.
View →~10M US with some retinopathy. Faricimab and longer-acting anti-VEGF; expanding role of earlier intervention in non-proliferative disease.
Diagnosis & staging, target-IOP setting, medical and laser therapy (SLT-first per LiGHT), MIGS and incisional surgery, angle-closure & secondary glaucomas, sustained-release delivery & progression monitoring.
View →~200K US across IRDs. Voretigene (Luxturna) for RPE65 disease; broad gene-therapy and optogenetic pipeline.
~1.5M+ US. Faricimab (dual Ang-2/VEGF) and high-dose aflibercept extend treatment intervals; port delivery system.
Diagnosis & empiric treatment (ESHRE 2022), hormonal suppression, GnRH antagonists/agonists with add-back & BMD monitoring, surgical excision, fertility & assisted reproduction, chronic pelvic pain & central sensitization, and adenomyosis — evidence tables.
View →~5M+ US. GLP-1 receptor agonists increasingly used for metabolic and reproductive features; phenotype-based management.
~1 in 25 US pregnancies. sFlt-1/PlGF ratio biomarker test (FDA-cleared 2023) for short-term risk prediction; aspirin prophylaxis.
~26M US (symptomatic in many). Oral GnRH antagonist combinations (relugolix-Myfembree, elagolix-Oriahnn); uterine-sparing options.
Pharmacotherapy algorithms, switching and augmentation strategies, TRD management, neuromodulation protocols, psychotherapy matching, and suicide risk assessment.
View → Available Now Clinical AlgorithmsAntipsychotic pharmacotherapy including KarXT/Cobenfy, clozapine optimization, LAI protocols, metabolic management, and guideline-aligned algorithms.
View →~1M+ US (lifetime). Higher-calorie refeeding evidence; olanzapine adjunct data; psilocybin and other novel-treatment trials.
Diagnosis & classification, acute mania & mixed states, bipolar depression, maintenance & relapse prevention, mood-stabilizer & antipsychotic safety and monitoring (lithium, valproate, lamotrigine, carbamazepine), suicide-risk management, and pregnancy & special populations — evidence-graded, CANMAT/ISBD- and NICE-concordant.
View →~2.5M US. Refined exposure-based therapy access; deep brain stimulation and TMS for refractory disease.
~6M US. Expanded buprenorphine access; high-dose and extended-release formulations; contingency management.
~13M US in a given year. Trauma-focused therapy evidence; MDMA-assisted therapy under regulatory review; stellate-ganglion-block research.
ICSD-3 classification, OSA severity and CPAP evidence, hypoglossal nerve stimulation, CBT-I protocols, insomnia pharmacotherapy, narcolepsy management, and RLS algorithms.
View →~600K US. Refined diagnostic criteria; intratympanic therapy; endolymphatic-hydrops imaging research.
~30M US (most undiagnosed). Tirzepatide (SURMOUNT-OSA, FDA Dec 2024) — first drug ever approved for OSA; hypoglossal nerve stimulation expanding.
~66K new/yr US. Time-sensitive corticosteroid therapy (oral and intratympanic); the 'ear emergency' message.
~1.2M US. Long-acting injectable cabotegravir/rilpivirine; lenacapavir (twice-yearly); lenacapavir PrEP (PURPOSE trials, FDA 2025).
~8-9K new/yr US; massive globally. Shorter all-oral regimens (BPaL/BPaLM) for drug-resistant TB; rapid molecular diagnostics.
Diagnosis & TRAb testing, antithyroid drug therapy & safety (agranulocytosis, PTU hepatotoxicity), radioactive iodine & thyroidectomy, beta-blockade, thyroid storm, Graves' orbitopathy, and pregnancy & special populations — evidence tables, ATA/ETA-concordant.
View → Available Now~15-20K new/yr US. Teprotumumab (Tepezza) — first drug to reduce proptosis without surgery; new IGF-1R agents in development.
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Clinical Reference → Available NowClinical management of beta-thalassemia for healthcare professionals. TIF 2025-aligned protocols for transfusion, MRI-guided iron chelation, luspatercept and mitapivat, HSCT including haploidentical with PTCy, and gene therapies Casgevy (exa-cel) and Zynteglo (beti-cel). Includes evidence tables, Utah resources, and international perspective.
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Clinical Reference → Available NowEvidence-based clinical reference on systemic lupus erythematosus for HCPs: 2019 EULAR/ACR classification criteria, 2025 ACR treatment guideline, treat-to-target with DORIS remission and LLDAS, hydroxychloroquine optimization with AAO 2025 retinal screening revision, biologic therapy selection (anifrolumab 4-year TULIP LTE data, subcutaneous autoinjector 2026; belimumab including pediatric LN autoinjector June 2025), lupus nephritis protocols (voclosporin AURORA, obinutuzumab REGENCY Phase III — FDA-approved October 2025, EULAR 2025 LN update), steroid minimization, NPSLE attribution, APS management, pregnancy optimization, CAR-T cell therapy (145-patient review ACR 2025, RESET-SLE rese-cel, allogeneic anti-CD19), deucravacitinib POETYK SLE Phase 3, cardiovascular risk, health equity, and Utah-specific referral resources.
Clinical Reference → Available NowEvidence-based clinical reference for endometrial cancer: TCGA molecular classification integrated into FIGO 2023 staging, surgical management with SLN mapping (FIRES trial), risk-adapted adjuvant therapy (PORTEC-3 10-year data, PORTEC-4a molecular profile-based), first-line immunotherapy-chemotherapy (dostarlimab RUBY OS data, pembrolizumab NRG-GY018 Nature Medicine 2025), lenvatinib-pembrolizumab 5-year KEYNOTE-775, HER2-directed therapy (T-DXd DESTINY-PanTumor02/Endometrial01), selinexor TP53wt maintenance, fertility-sparing protocols (ESGO/ESHRE/ESGE 2023), RAINBO molecular-guided adjuvant program, Lynch syndrome universal MMR screening, NCCN v3.2025, and Utah referral resources.
Clinical Reference → Available NowEvidence-based clinical reference for type 1 diabetes: autoantibody staging (IAA, GAD65, IA-2, ZnT8), insulin formulation comparison, AID system selection (Omnipod 5, Control-IQ, 780G), teplizumab prescribing (Tzield, expanded to age 1+), glycemic targets by population, complication screening protocols, Vertex VX-880 stem cell therapy pipeline, ADA 2026 Standards of Care, ISPAD 2024, and Utah referral resources.
Clinical Reference → Available NowEvidence-based clinical reference for balance and dizziness disorders: HINTS exam, vestibular testing interpretation, BPPV repositioning techniques, Meniere's treatment ladder, vestibular migraine criteria, PPPD management, rehabilitation protocols, acoustic neuroma surveillance, and falls prevention.
Clinical Reference → Available NowEvidence-based clinical reference for AL amyloidosis: diagnostic algorithms, amyloid typing, organ staging, treatment selection, response monitoring, emerging therapies, and guideline-concordant protocols.
Clinical Reference → Available NowClinical reference for transient neurological events: TGA/TEA/TIA differential diagnosis, ABCD2 risk stratification, neuroimaging protocols, secondary prevention, and cognitive follow-up.
Clinical Reference → Available NowClinical reference for autism spectrum disorder: DSM-5-TR criteria, screening tools, behavioral interventions, pharmacotherapy for co-occurring conditions, genetic evaluation, and lifespan management protocols.
Clinical Reference → Available NowClinical reference for PCOS and premature ovarian insufficiency: Rotterdam criteria, pharmacotherapy, fertility algorithms, HRT protocols, cardiometabolic monitoring, and guideline concordance for healthcare professionals.
Clinical Reference → Available NowClinical reference for ADHD and OCD: DSM-5-TR criteria, stimulant and non-stimulant pharmacotherapy, SSRI dosing for OCD, CBT/ERP protocols, comorbidity management, treatment-resistant algorithms, and guideline concordance.
Clinical Reference → Available NowClinical reference for asthma: stepwise management aligned with GINA 2025/2026, phenotype-directed biologic selection, MART/SMART therapy, acute exacerbation protocols, inhaler device selection, and monitoring/step-down algorithms.
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