Duchenne News

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)...

On November 20, 2025, Avidity Biosciences shared a Community Letter detailing their definitive merger acquisition by Novartis. This letter comes after the official announcement on October 26, 2025.  To view...

On November 19, 2025, Avidity Biosciences, Inc. announced its Managed Access Program (MAP) for investigational therapy delpacibart zotadirsen (del-zota) for eligible people with Duchenne muscular dystrophy mutations amenable to exon...

On November 16, 2025, Sarepta Therapeutics Inc. shared a Community Letter with updates regarding the label for ELEVIDYS, Sarepta’s approved gene therapy for Duchenne muscular dystrophy. To view Sarepta’s Community...

On November 6, 2025, Entrada Therapeutics reported financial results for the third quarter ended September 30, 2025, and highlighted recent business updates. Please continue reading to view their full press...

On November 3, 2025, Solid Biosciences Inc. reported financial results for the third quarter ended September 30, 2025, and announced positive new interim data from the Phase 1/2 INSPIRE DUCHENNE clinical...