Duchenne News

On February 17, 2026, Entrada Therapeutics, Inc. announced that an independent Data Monitoring Committee (DMC), per study protocol, has reviewed all available safety and PK data from the eight patients...

On February 12, 2026, PTC Therapeutics, Inc. announced that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy...

On February 12, 2026, Satellos Bioscience Inc, announced that the first participant has been dosed in BASECAMP, a three-month, randomized, double-blind, placebo-controlled, proof-of-concept, Phase 2 pediatric study of SAT-3247 for...

On January 22, 2026, Avidity Bioscience shared a Managed Access Program (MAP) – Frequently Asked Questions sheet with the Duchenne community to provide clarity and address the most frequently asked...

On January 13, 2026, Solid Biosciences Inc. announced it will provide a corporate update outlining progress in advancing its neuromuscular and cardiac gene therapy programs in a presentation delivered by...

On December 16, 2025, The U.S. Department of Health and Human Services (HHS) Secretary Robert F. Kennedy, Jr. approved the addition of Duchenne Muscular Dystrophy (DMD) and Metachromatic Leukodystrophy (MLD)...

On December 1, 2025, Upsher-Smith Laboratories, LLC (Upsher-Smith) announced the launch of KYMBEE™ (deflazacort) Tablets, indicated for the treatment of Duchenne muscular dystrophy in patients 5 years of age and...

On December 8, 2025, Dyne Therapeutics, Inc. announced positive topline results from the Registrational Expansion Cohort (REC) of its Phase 1/2 DELIVER trial evaluating zeleciment rostudirsen (z-rostudirsen, also known as...

On November 25, 2025, Sarepta Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved dosing in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort...

On November 24, 2025, Wave Life Sciences announced they have initiated Part C of FORWARD-53, a clinical trial evaluating the investigational molecule WVE-N531 in boys with Duchenne muscular dystrophy (DMD)...