News

A second person with Duchenne muscular dystrophy (DMD) has died of acute liver failure after being treated with the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl). A first death was announced in March and both occurred in people with DMD who were no longer able to walk. Following…

The European Comission has conditionally approved Duvyzat (givinostat) to be taken alongside corticosteroids for treating people who are 6 and older with Duchenne muscular dystrophy (DMD). “This milestone means that a broad range of patients with DMD have access to a new treatment,” Francesco De Santis, president of…

The U.S. Food and Drug Administration (FDA) granted platform technology designation to the viral vector used in SRP-9003, an investigational gene therapy for limb-girdle muscular dystrophy type 2E (LGMD2E). “This is one of the first programs to receive platform technology designation and an important recognition by FDA of…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to ISX9-CPC, IPS Heart’s experimental stem cell therapy for heart problems in Duchenne muscular dystrophy (DMD), the company announced in an email sent to Muscular Dystrophy News Today. This FDA designation aims to accelerate the development…

Following lackluster data from a Phase 2 clinical trial, Pepgen will discontinue developing PGN-ED051, its experimental exon 51-skipping therapy for Duchenne muscular dystrophy (DMD). The company said it will wind down all DMD-related research and development activities, focusing instead on an investigational therapy for myotonic dystrophy type…

CureDuchenne is investing $1 million in Entos Pharmaceuticals to support the development of a new gene therapy for Duchenne muscular dystrophy (DMD) that aims to overcome the limitations of existing gene therapies. “This investment underscores our continued use of venture philanthropy to catalyze progress [toward] transformative treatments…

Satellos Bioscience’s SAT-3247 was safe, well-tolerated, and showed initial signs of increasing muscle strength in five adults with Duchenne muscular dystrophy (DMD), the company said. “We believe the findings from this Phase 1b study support our plan to advance SAT-3247 into a placebo-controlled Phase 2 trial,” Satellos CEO…

An investigational gene therapy called GNT0004 has demonstrated sustained stabilization of motor function at up to two years of follow-up in two boys with Duchenne muscular dystrophy (DMD). The boys received what was determined to be the effective dose of GNT0004, which was developed by Genethon, during the initial…

The one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) was associated with stabilizations in motor function for boys with Duchenne muscular dystrophy (DMD) who were treated when they were 8 to 9 years old, according to new trial analyses. Boys with DMD at this age are typically expected to show…

Note: This story was updated May 19, 2025, to clarify the requirements needed for Elevidys to transition to full approval. The Japanese Ministry of Health, Labour, and Welfare (MHLW) has conditionally approved the gene therapy Elevidys (delandistrogene moxeparvovec-rokl) to treat some cases of Duchenne muscular dystrophy (DMD). The…