The 23rd International Conference on Duchenne and Becker Muscular Dystrophy recently took place, focusing on advancements and challenges in the field. This event highlights ongoing efforts to improve awareness and support for affected individuals and families.

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.

NORD commends HHS for including metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD) in the Recommended Uniform Screening Panel, enhancing early detection for these rare diseases. This decision supports better patient outcomes through timely diagnosis.

The U.S. Department of Health and Human Services has officially added Duchenne muscular dystrophy and Metachromatic Leukodystrophy to the Federal Recommended Uniform Screening Panel. This decision enhances early detection and intervention for these rare diseases, impacting newborn screening practices nationwide.