Duchenne muscular dystrophy
Therapeutic area
Neurology
Rare disease
Yes
- 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
- 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
- Severity
- Severe
- Genetic
- Hereditary
Sources for recorded values
- Neurology
- Neuromuscular Disease
- Rheumatology
Sources for recorded values
- MONDO ID
- 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
- Hereditary neurological disease
- Hereditary neuromuscular disease
- Hereditary skeletal muscle disorder
- Human disease
- Muscle tissue disorder
- Muscular dystrophy
- Musculoskeletal system disorder
- Myopathy
- Nervous system disorder
- 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 Phase 1
- Flavocoxid Phase 1
Other linked drugs (2)
- Rituximab
- Testosterone