The post 23rd International Conference on Duchenne and Becker Muscular Dystrophy appeared first on EURORDIS-Rare Diseases Europe.

Original title: “23rd International Conference on Duchenne and Becker Muscular Dystrophy”

After a spate of patient deaths in 2025 linked to the company’s Duchenne gene therapy, Sarepta shared new data showing benefits of the therapy three years after dosing. In an investor call to discuss the second mortality, CEO Doug Ingram insisted that “liver injury is a known risk of AAV therapies.” · Still, the deaths ushered in months of regulatory difficulties and business realignment efforts for Sarepta. In July 2025, the biotech laid off around 500 employees in a pivot that included moving away from gene therapies and leaning into its siRNA pipeline. The benefits of taking Elevidys, Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy, continue three years post treatment, the company said Monday. Monday’s readout comes after a tumultuous 2025 for Elevidys. In March, the gene therapy was implicated in the death of a young boy with DMD who developed acute liver injury leading to liver failure. Get daily news updates when you subscribe to GenePool!

Original title: “Sarepta’s DMD Gene Therapy Staves Off Disease Three Years After Treatment - BioSpace”

HRSA published a Federal Register notice on August 14, 2025 (90 FR 39197), requesting comments from the public on the potential recommendation of adding Duchenne Muscular Dystrophy (DMD) to the Recommended Uniform Screening Panel (RUSP). After consideration of public comments and evidence-based reports, HRSA recommended to the HHS Secretary that DMD be added to the RUSP. The Secretary has accepted the recommendation as detailed in this notice. Conditions listed on the RUSP are part of the eviden

Original title: “Addition of Duchenne Muscular Dystrophy to the Recommended Uniform Screening Panel”

The National Organization for Rare Disorders (NORD) applauds the Department of Health and Human Services (HHS) for adding metachromatic leukodystrophy (MLD) and Duchenne muscular dystrophy (DMD) to the Recommended Uniform … Continue reading "NORD CEO Statement on the Addition of Metachromatic Leukodystrophy (MLD) and Duchenne Muscular Dystrophy (DMD) to the Recommended Uniform Screening Panel"

Original title: “NORD CEO Statement on the Addition of Metachromatic Leukodystrophy (MLD) and Duchenne Muscular Dystrophy (DMD) to the Recommended Uniform Screening Panel”

The U.S. Department of Health and Human Services has added Duchenne muscular dystrophy and Metachromatic Leukodystrophy (MLD) to the federal Recommended Uniform Screening Panel (RUSP)! The RUSP is a list of disorders […] The post HHS Adds Duchenne Muscular Dystrophy and MLD to the Federal Recommended Uniform Screening Panel (RUSP) appeared first on EveryLife Foundation for Rare Diseases.

Original title: “HHS Adds Duchenne Muscular Dystrophy and MLD to the Federal Recommended Uniform Screening Panel (RUSP)”