Keros to Start Rinvatercept DMD Trial in Q1 2026, Plans ALS Regulatory Talks
Keros Therapeutics plans a phase II DMD trial of rinvatercept in Q1 2026 and regulatory talks for ALS in H2 2026. Cash stands at $383 million with runway into H1 2028.
DMD
DiseaseDisease Profile
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.
- Category
- Neuromuscular disorder (muscular dystrophy)
- Prevalence
- Approximately 1 in 3,500 to 5,000 male births
- G71.01
Related News
EV-Based Gene Therapy Delivers Full-Length Dystrophin mRNA, Restores Muscle in DMD Models
Researchers at MD Anderson developed an extracellular vesicle-based platform that delivers full-length DMD mRNA, restoring dystrophin production and improving muscle function in preclinical Duchenne muscular dystrophy models. The study, published in Nature Biomedical Engineering, showed reduced side effects compared with viral gene therapies.
Deramiocel Slows Upper Limb Decline in Duchenne Muscular Dystrophy; FDA Review Ongoing
Phase III data show deramiocel slowed upper limb decline by 54% in Duchenne muscular dystrophy. The FDA is re-evaluating the BLA with HOPE-3 results, with a submission of the clinical study report expected in February 2026.
FDA Grants Rare Pediatric Disease Designation to Atossa's (Z)-Endoxifen for Duchenne Muscular Dystrophy
The FDA granted Rare Pediatric Disease designation to Atossa Therapeutics' (Z)-endoxifen for treating Duchenne Muscular Dystrophy. The designation may qualify the drug for a valuable Priority Review Voucher upon approval. Atossa reported a net loss of $34.8 million for 2025 as it advances clinical development.
Federal Circuit Revives Regenxbio Patent Suit Against Sarepta Over DMD Gene Therapy
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 and Satellos advance Duchenne muscular dystrophy treatment trials
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.
DMD Care Expands With Multidisciplinary Management, Earlier Pathology Focus
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 Advances SGT-003 DMD Gene Therapy with FDA Alignment, Reports No Liver Toxicity
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.
FDA Grants Fast Track Designation to Precision BioSciences' DMD Gene Therapy
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 Receives FDA Fast Track Designation for PBGENE-DMD Gene Editing Therapy
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.
Related Clinical Trials
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07766980 |
Evaluating VM100 Nutritional Supplement for Improving Quality of Life in Duchenne Muscular Dystrophy Patients |
RECRUITING | NA |
| NCT07608432 |
Efficacy, Safety, and Tolerability of Zeleciment Rostudirsen (DYNE-251) Administered Intravenously Every 4 Weeks in Ambulatory Participants With Duchenne Muscular Dystrophy (FORZETTO) |
RECRUITING | PHASE3 |
| 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 |
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 |