Duchenne News

Precision BioSciences has dosed the first patient at Arkansas Children’s Hospital, a PPMD Certified Duchenne Care Center and designated MDA Care Center, in the Phase 1/2 Function-DMD clinical trial evaluating...

Entrada Therapeutics has shared an update with the Duchenne community on the continued progress of its ELEVATE-44-201 and ELEVATE-45-201 clinical studies. This includes revised timelines for study data readouts, now...

Satellos recently attended the International Congress on Neuromuscular Diseases (ICNMD) in Florence, Italy (July 7–11), where they presented six-month interim data from their ongoing TRAILHEAD clinical study evaluating SAT-3247 in...

  On July 20, Dyne Therapeutics announced that the FDA has accepted for review the Biologics License Application (BLA) for zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251) for the treatment...

On June 25, 2026, Avidity Biosciences, Inc. shared that they have submitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of delpacibart zotadirsen (also known as...

The FDA has scheduled an Advisory Committee meeting for July 29, 2026 to review Deramiocel, Capricor’s investigational cell therapy for Duchenne muscular dystrophy. The meeting will be held in person with the...

On June 30, 2026, Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for filing the supplemental New Drug Applications (sNDAs) for AMONDYS 45® (casimersen)...

On June 29, 2026, Satellos Bioscience Inc., announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SAT-3247 for the treatment of Duchenne. Please read...

On June 26, 2026, Solid Biosciences, Inc. shared a Letter to the Duchenne Community containing updates on the INSPIRE DUCHENNE and Phase 3 IMPACT DUCHENNE clinical studies, regulatory engagement, and...

On June 24, 2026, REGENXBIO Inc. announced the successful completion of dosing in the confirmatory study of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy. They also shared...