DMD usually refers to Duchenne muscular dystrophy, a progressive X-linked recessive neuromuscular disorder caused by mutations in the DMD gene leading to loss of functional dystrophin. It presents in early childhood and primarily affects males, with progressive muscle degeneration and weakness over time. The disease is one of the dystrophinopathies.
A U.S. appeals court revived Regenxbio’s patent suit against Sarepta, holding the asserted gene-therapy host cell claims are markedly different from anything occurring in nature.
Mesoblast received FDA clearance for a registrational DMD trial of remestemcel-L-rknd, while Satellos began dosing boys in the Phase 2 BASECAMP study of SAT-3247. The studies are enrolling children ages 5-9 and 7-9, respectively.
Duchenne muscular dystrophy care is shifting toward broader multidisciplinary management and closer attention to early pathology, biomarkers, and treatment timing. Clinicians described an expanding treatment landscape and identified neurology, cardiology, pulmonary, and endocrinology as core priorities.
Solid Biosciences has secured FDA alignment on a registration pathway for its Duchenne muscular dystrophy gene therapy SGT-003, with 36 patients dosed to date showing no drug-induced liver injury, myocarditis, or other serious adverse events reported with competing therapies.
Precision BioSciences receives FDA Fast Track designation for PBGENE-DMD, a gene editing therapy for Duchenne muscular dystrophy designed to treat patients with mutations in exons 45-55.
Precision BioSciences announced FDA Fast Track designation for PBGENE-DMD, a first-in-class gene editing therapy for Duchenne muscular dystrophy targeting mutations in exons 45-55, representing up to 60% of DMD patients.
Artificial intelligence is revolutionizing rare disease diagnosis, cutting diagnostic timelines from years to weeks. Rare Disease Day on February 28 highlights challenges faced by over 300 million people worldwide living with more than 7,000 distinct rare conditions.
Chugai Pharmaceutical launched ELEVIDYS in Japan as the first regenerative medical product for Duchenne muscular dystrophy following its listing on the National Health Insurance reimbursement price list on February 20, 2026.
Japan's Ministry of Health, Labour and Welfare granted Orphan Drug designation to Dyne Therapeutics' investigational treatment zeleciment-basivarsen for myotonic muscular dystrophy type 1, adding to existing designations in the U.S. and Europe.
PTC Therapeutics has withdrawn its New Drug Application resubmission for ataluren (Translarna) for nonsense mutation Duchenne muscular dystrophy after the FDA indicated the submission lacked substantial evidence of effectiveness.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07587242 |
A Phase 3 Study to Evaluate the Safety and Efficacy of AOC 1044 (Also Referred to as Delpacibart Zotadirsen) in Participants With DMD With Gene Mutations Amenable to Exon 44 Skipping |
NOT_YET_RECRUITING | PHASE3 |
| NCT07287189 |
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients |
RECRUITING | PHASE2 |
| NCT07058662 |
A Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of BBM-D101 in the Treatment of Duchenne Muscular Dystrophy. |
RECRUITING | PHASE1/PHASE2 |
| NCT07015632 |
ML-Based Multi-Sensor Fall Risk Screening in DMD |
RECRUITING | |
| NCT06581887 |
Defining Outcome Measures for Behavioural and Emotional Problems in Dystrophinopathies |
RECRUITING | |
| NCT06290713 |
Vasodilator and Exercise Study for DMD (VASO-REx) |
RECRUITING | PHASE2 |
| NCT06244082 |
Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping |
ACTIVE_NOT_RECRUITING | PHASE2 |
| NCT05996003 |
NS-089/NCNP-02-201 in Boys With Duchenne Muscular Dystrophy (DMD) |
RECRUITING | PHASE2 |
| NCT05635266 |
Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives |
RECRUITING | |
| NCT05102916 |
Swiss Registry for Neuromuscular Disorders |
RECRUITING |
