Duchenne News

On March 11, 2026, Solid Biosciences Inc. reported updated positive interim data from the ongoing Phase 1/2 INSPIRE DUCHENNE clinical trial of SGT-003. They shared these updates in both a...

On March 11, 2026, REGENXBIO Inc. announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial of RGX-202, a potential best-in-class gene therapy for Duchenne muscular dystrophy. They...

On March 11, 2026, ITF Therapeutics shared a Community Letter outlining new data and analyses presented on DUVYZAT® (givinostat) at 2026 MDA Clinical and Scientific Conference. To view their Community...

On March 10, 2026, Edgewise Therapeutics, Inc. presented at the 2026 MDA Clinical and Scientific Conference, long-term data from its MESA open-label extension study of sevasemten in Becker muscular dystrophy...

On March 8, 2026, Dyne Therapeutics, Inc. (Nasdaq: DYN) announced additional positive data from the ongoing Phase 1/2 DELIVER clinical trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), in...

On February 26, 2026, Entrada Therapeutics, Inc. reported financial results for the fourth quarter and full year ended December 31, 2025, and highlighted recent business updates. This full release can...

On February 20, 2026, Roche shared an important update in a community letter regarding their work in bone health in Duchenne and the SHIELD DMD study. Read more by visiting...

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On February 12, 2026, PTC Therapeutics, Inc. announced that it has withdrawn the New Drug Application (NDA) resubmission for Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy...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