Comprehensive, easy-to-understand information about this condition
How we create this content →Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Helpful links for rare disease information and support
Questions that may be helpful when speaking with your healthcare team
Research studies investigating treatments and therapies for this condition.
Active Trials
Total Trials
Data from ClinicalTrials.gov Feb 5, 2026
Common questions about DMD-related muscular dystrophy
AI-Generated Content: This summary was generated using AI. Always consult with qualified healthcare providers for medical guidance.
Kisho delivers this disease record via API, including phenotypes (HPO), genes, orphan drug designations, screening status, and PAG mapping, with version history and governance.
AI-curated news mentioning DMD-related muscular dystrophy
Updated Feb 9, 2026
Avidity Biosciences, REGENXBIO, and Capricor Therapeutics are preparing key regulatory applications for Duchenne muscular dystrophy treatments this year, signaling a potential shift in the DMD pipeline following Sarepta's recent challenges. CureDuchenne's CSO Michael Kelly notes a growing momentum in the field.
Sarepta Therapeutics reports that its gene therapy Elevidys for Duchenne muscular dystrophy shows continued benefits three years post-treatment, despite facing regulatory challenges and a significant workforce reduction. The therapy's efficacy comes amid scrutiny following patient deaths linked to liver injury, highlighting the risks associated with AAV therapies.
Regenxbio's RGX-202 gene therapy for Duchenne muscular dystrophy (DMD) demonstrated significant improvements in patient outcomes, exceeding expected disease progression at 12 and 18 months post-treatment in a pivotal trial. The company plans to submit a Biologics License Application (BLA) in mid-2026 via an accelerated approval pathway.
The HHS Secretary has accepted the recommendation to add Duchenne Muscular Dystrophy (DMD) to the Recommended Uniform Screening Panel (RUSP), following public comments and evidence-based reports. This decision could enhance early detection and intervention for DMD, impacting patient outcomes significantly.