Marisa Wexler, MS,  senior science writer—

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…

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…

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…

The U.S. Food and Drug Administration (FDA) is on track to make a decision about deramiocel, an experimental cell therapy designed to treat heart disease in Duchenne muscular dystrophy (DMD), by the end of August. The FDA agreed this year to review an application to approve deramiocel for…

It’s been nearly two years since Elevidys (delandistrogene moxeparvovec-rokl), a gene therapy for Duchenne muscular dystrophy (DMD) developed by Sarepta Therapeutics, was approved by the U.S. Food and Drug Administration (FDA), marking the first time a gene therapy for DMD was authorized in the U.S. Since its…

The U.S. Food and Drug Administration (FDA) has cleared Sarepta Therapeutics to start dosing and screening in a first clinical trial testing the gene therapy SRP-9005 in people with limb-girdle muscular dystrophy type 2C (LGMD2C). Sarepta said advancement continues for SRP-9003 and SRP-9004, two gene therapies…

KER-065, a potential treatment for Duchenne muscular dystrophy (DMD) and other neuromuscular diseases, showed a good safety profile in a Phase 1 clinical trial. No serious side effects were seen in the trial, which tested single and multiple doses of KER-065 in healthy volunteers, and no major safety issues…

The use of experimental gene therapy BB-301 led to improvements in swallowing ability for the first three people with oculopharyngeal muscular dystrophy (OPMD) — a type of muscular dystrophy marked by muscle weakening in the throat and eyes — in a clinical trial. That’s according to interim data…

Long-term treatment with Duvyzat (givinostat) may help delay the loss of walking ability and lessen the decline in lung function for boys with Duchenne muscular dystrophy (DMD), new analyses indicate. The studies were presented in a series of posters at this year’s meeting of the Muscular Dystrophy…