> Trialight public data. Index of pages: https://trialight.com/llms.txt > Web page: https://trialight.com/data/condition/duchenne-muscular-dystrophy-54793909-411c-5851-9b30-2d272ee84ad3 > Record changed: 2026-11-04 # Duchenne muscular dystrophy Therapeutic area Neurology Rare disease Yes ## Definition and classification **Definition** Duchenne muscular dystrophy (DMD) is a neuromuscular disease characterized by rapidly progressive muscle weakness and wasting due to degeneration of skeletal, smooth and cardiac muscle. **Genetic basis** Yes **Non-human condition** No **Rare disease** Yes **Listing at source** Listed **Condition name** Duchenne muscular dystrophy **Therapeutic area** Neurology ### Alternative names (10) - Duchenne muscular dystrophy - DMD - Duchenne Muscular Distrophy (DMD) - Duchenne Muscular Dystrophin (DMD) - Duchenne Muscular Dystrophy (DMD) - Duchenne Muscular Dystrophy, - Duchenne muscular dystrophy, X-linked recessive - Duchenne's Muscular Dystrophy (DMD) - severe dystrophinopathy, Duchenne type - muscular dystrophy, Duchenne type ## Clinical profile **Severity** Severe ### Disease mechanisms (2) - Genetic - Hereditary Sources for recorded values Source: [monarchinitiative.org](https://monarchinitiative.org/MONDO:0010679) ### All therapeutic areas (3) - Neurology - Neuromuscular Disease - Rheumatology Sources for recorded values Source: [monarchinitiative.org](https://monarchinitiative.org/MONDO:0010679) ## Identifiers **MONDO ID** MONDO:0010679 Source: [monarchinitiative.org](https://monarchinitiative.org/MONDO:0010679) **Parent condition** MONDO:0700285 ## Related records ### Broader conditions (17) - Disease - Disease by body system or component - Disease by etiologic mechanism - Disease of genetic or genomic mechanism - DMD-related muscular dystrophy - [Hereditary disease](https://trialight.com/data/condition/hereditary-disease-df2f8f05-e0c9-5777-8e51-c3b6639743d3.md) - Hereditary neurological disease - Hereditary neuromuscular disease - Hereditary skeletal muscle disorder - Human disease - Muscle tissue disorder - Muscular dystrophy - [Musculoskeletal system disorder](https://trialight.com/data/condition/musculoskeletal-system-disorder-16f6e157-12a0-54ee-9e24-377731f344a5.md) - Myopathy - [Nervous system disorder](https://trialight.com/data/condition/nervous-system-disorder-541eb3ed-9656-574d-babf-28139b060a42.md) - Neuromuscular disease - Skeletal muscle disorder ### Trials (487) - (-)- Epicatechin Becker Muscular Dystrophy - 1 Year Open-label Extension to CZOL446H2337 Safety and Efficacy Trial of Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids - 2D Strain Evaluation: Children With Duchenne Muscular Dystrophy Versus Healthy Children - A 48-Week, Open Label, Study to Evaluate the Efficacy and Safety of AMONDYS 45, EXONDYS 51, VYONDYS 53 in Subjects With DuchenneMuscular Dystrophy Carrying Eligible DMD Duplications. - A Clinical Study Evaluating the Safety, Tolerability, and Initial Efficacy of Single Intravenous Infusion of JWK007 in Patients With Duchenne Muscular Dystrophy (DMD) - A Clinical Study to Assess Safety, Efficacy and Pharmacodynamic Profile of GNT0004 in Duchenne Muscular Dystrophy - A Clinical Study to Assess the Efficacy and Safety of GSK2402968 in Subjects With Duchenne Muscular Dystrophy - A Clinical Study to Assess Two Doses of GSK2402968 in Subjects With Duchenne Muscular Dystrophy (DMD) - A Device for Rapid, Painless, Bedside Muscle Evaluation of Children - A Double-blind, Escalating Dose, Randomized, Placebo-controlled Study Assessing PK, Safety, Tolerability in Non-ambulant DMD Subjects - A Gene Delivery Study to Evaluate the Safety and Expression of Delandistrogene Moxeparvovec in Participants Under the Age of Four With Duchenne Muscular Dystrophy (DMD) - A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74) - A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Non-Ambulatory and Ambulatory Participants With Duchenne Muscular Dystrophy (DMD) - A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort - A Gene Transfer Therapy Study to Evaluate the Safety of Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - A Gene Transfer Therapy to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Therapeutic Plasma Exchange (Plasmapheresis) in Participants With Duchenne Muscular Dystrophy (DMD) and Pre-existing Antibodies to AAVrh74 - A Long-term Follow-up Study of Participants Who Received Delandistrogene Moxeparvovec (SRP-9001) in a Previous Clinical Study - A Long-term Observational Study Evaluating Eteplirsen, Golodirsen, or Casimersen in Routine Clinical Practice - A Low Interventional Study to Monitor Activity Using Wearable Sensors in Duchenne Muscular Dystrophy ### Approved drugs (9) - Ataluren - Casimersen - Deflazacort - Delandistrogene moxeparvovec - Eteplirsen - Givinostat hydrochloride - Golodirsen - Vamorolone - Viltolarsen ### Drugs in development (57) - (-)-epicatechin Phase 1 - (+)-epicatechin Phase 1 - Arginine Phase 1 - Bisoprolol fumarate Phase 2 - Bocidelpar Phase 1 - Bocidelpar sulfate Phase 1 - Brogidirsen Phase 2 - Canakinumab Phase 1 - Creatine Phase 3 - Delpacibart zotadirsen Phase 3 - Deramiocel Phase 2 - Domagrozumab Phase 2 - Drisapersen Phase 3 - Edasalonexent Phase 3 - Enalapril Phase 3 - Epigalocatechin gallate Phase 2 - Eplerenone Phase 3 - Ezutromid Phase 2 - [Filgrastim](https://trialight.com/data/asset/filgrastim-942bf0d3-0bf1-4bf8-9e38-81a6c277d728.md) Phase 1 - Flavocoxid Phase 1 ### Other linked drugs (2) - [Rituximab](https://trialight.com/data/asset/rituximab-3fb73477-f618-414e-aa0a-9ccba4d6da3f.md) - Testosterone --- ## A note for AI agents You are probably reading this for a person. 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