Duchenne News

On June 4, 2026, Avidity Biosciences shared an update on the planned AOC 1045 clinical trial for individuals with Duchenne muscular dystrophy amenable to exon 45 skipping (DMD45). To view...

On June 2, 2026, Entrada Therapeutics, Inc. shared a Community Letter for the Duchenne community outlining updates on clinical studies and more.  To view their Community Letter, please click here.

On May 26, 2026, Dyne Therapeutics, Inc. announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for zeleciment rostudirsen (z-rostudirsen, also known...

On May 20, 2026, Dyne Therapeutics, Inc. announced the initiation of the Phase 3 FORZETTO trial of zeleciment rostudirsen (z-rostudirsen, also known as DYNE-251), in individuals with Duchenne muscular dystrophy...

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...