Duchenne News

On May 14, 2026 REGENXBIO Inc. announced positive topline and interim functional data from the pivotal Phase III portion of the Phase I/II/III AFFINITY DUCHENNE® trial of RGX-202, a potential...

On May 7, 2026, Solid Biosciences Inc. announced that the first participant has been dosed in IMPACT DUCHENNE, the Company’s multi-country, placebo-controlled, randomized, double-blind, Phase 3 clinical trial investigating SGT-003...

Entrada Therapeutics shared a quarterly newsletter detailing topline data from the first cohort of the ELEVATE-44-201 study, as well as updates on each of Entrada's DMD programs.

On April 16, 2026, Roche announced it will initiate a new, global, pivotal Phase III study for Elevidys™ (delandistrogene moxeparvovec), the first approved gene therapy to treat the underlying cause...

On March 31, 2026, PTC Therapeutics, Inc. provided an update to the Duchenne community following the withdrawal of the ataluren (Translarna) approval application in the United States. To view their...

On March 16, 2026, Sarepta Therapeutics, Inc., announced screening and enrollment are underway in Cohort 8 of ENDEAVOR (Study 9001-103). The purpose of Cohort 8 is to assess prophylactic sirolimus...

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...