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.
Duchenne Muscular Dystrophy
DiseaseAlso known as: nonsense mutation DMD
Disease Profile
Duchenne muscular dystrophy is a progressive X-linked recessive neuromuscular disorder caused by pathogenic variants in the DMD gene, leading to dysfunctional or absent dystrophin. It usually presents in early childhood and predominantly affects males, with progressive muscle weakness and degeneration. It is one of the most common inherited neuromuscular disorders of childhood.
- Category
- Neuromuscular disorder (muscular dystrophy)
- Prevalence
- Approximately 1 in 3,500 to 5,000 male births
- G71.01
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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 |
| NCT07704099 |
Safety and Efficacy of KER-065 in Participants With Duchenne Muscular Dystrophy |
NOT_YET_RECRUITING | PHASE2 |
| NCT07673809 |
A Study to Evaluate the Tolerability, Safety and Efficacy of GNR-097 Gene Therapy in Pediatric Patients With Duchenne Muscular Dystrophy |
RECRUITING | PHASE1/PHASE2 |
| NCT07542314 |
Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting |
NOT_YET_RECRUITING | PHASE4 |
| NCT07287189 |
Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients |
RECRUITING | PHASE2 |
| NCT07209332 |
Open-Label Extension Study of WVE-N531 in Patients With Duchenne Muscular Dystrophy |
ENROLLING_BY_INVITATION | PHASE2 |
| NCT07172737 |
Investigation of the Effects of Functional Inspiratory Muscle Training Applied With Tele-Rehabilitation on Respiratory and Functional Parameters and Quality of Life in Children With Duchenne Muscular Dystrophy: Randomized Controlled Study |
COMPLETED | NA |
| NCT07160634 |
A Study of SGT-003 Gene Therapy in Ambulant Males With Duchenne Muscular Dystrophy (IMPACT DUCHENNE) |
RECRUITING | PHASE3 |
| NCT06868784 |
Investigation of the Relationship Between Executive Functions and Occupational Performance of Children With Duchenne Muscular Dystrophy |
ENROLLING_BY_INVITATION | |
| NCT06861270 |
Estimation of Non-Reimbursable Costs for Patients With Duchenne Muscular Dystrophy in France |
COMPLETED |