Duchenne News

On June 26, 2025, Edgewise Therapeutics, Inc. unveiled positive results in its sevasemten program for Becker and Duchenne muscular dystrophies. Please continue reading to view their full press release. BOULDER, Colo., June...

On June 24, 2025, Capricor Therapeutics provided regulatory updates related to its Biologics License Application (BLA) for Deramiocel, the Company’s lead cell therapy candidate for the treatment of Duchenne Muscular...

On June 11, 2025, Capricor Therapeutics announced the successful completion of the U.S. Food and Drug Administration’s (FDA) Pre-License Inspection (PLI) of its SanDiego manufacturing facility for Deramiocel, the Company’s...

On June 9, 2025, Avidity Biosciences, Inc. announced positive topline data from the dose escalation cohorts of the delpacibart braxlosiran (del-brax) Phase 1/2 FORTITUDE™ program in Facioscapulohumeral Muscular Dystrophy (FSHD)....

On June 9, Avidity Biosciences Inc. announced the accelerated approval regulatory pathway in the United States is open for delpacibart braxlosiran (del-brax) in the treatment of facioscapulohumeral muscular dystrophy (FSHD). Additionally, the...

Updates include positive functional, safety and biomarker data for RGX-202, REGENXBIO's potential best-in-class, investigational gene therapy for Duchenne muscular dystrophy.

On May 28, 2025, PepGen announced that based on the levels of dystrophin protein measured in the 10 mg/kg cohort of its CONNECT1-EDO51 study investigating PGN-EDO51 in Duchenne muscular dystrophy...

On May 28, 2025, Entrada Therapeutics, Inc. announced it has received authorization from the Health Authorities and Ethics Committees of multiple countries under the European Union Clinical Trial Regulation (EU-CTR)...

On May 8, 2025, Entrada Therapeutics reported financial results for the first quarter ended March 31, 2025, and highlighted recent business updates. Please continue reading to view their full press...

On April 25, 2025, ITF Therapeutics announced that the Committee for Medicinal Products (CHMP) of the European Medicines Agency (EMA) recommended granting a conditional marketing authorization for DUVYZAT® (givinostat) for...