News

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…

The Japan Ministry of Health, Labour and Welfare (MHLW) has granted orphan drug status to delpacibart etedesiran, known as del-desiran, an investigational therapy from Avidity Biosciences now in clinical testing for myotonic dystrophy type 1 (DM1). This designation is intended to support the development of therapies for rare…

Health Canada has agreed to review an application by Kye Pharmaceuticals seeking the approval of vamorolone for treating Duchenne muscular dystrophy (DMD) — and granted it priority review. Priority review shortens the review period from the standard 10 months to six. It’s given to therapies that have the…

An independent data monitoring committee (DMC) favors continuing dosing Elevidys (delandistrogene moxeparvovec-rokl) to people with Duchenne muscular dystrophy (DMD) in ongoing clinical trials, according to developer Sarepta Therapeutics. The determination comes after recruitment and dosing in certain clinical trials were paused after a young man who…

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…

Patient advocate Donavon Decker has been given the 2025 MDA Legacy Award for Community Impact in Research, recognizing his efforts across decades on behalf of the muscular dystrophy community. Decker, who has limb-girdle muscular dystrophy (LGMD) type 2D, was honored at the Muscular Dystrophy Association (MDA)’s annual…

Treatment with DYNE-101 is leading to improvements in muscle function — with notable gains in finger function — among people with myotonic dystrophy type 1 (DM1) in a Phase 1/2 clinical trial, according to updated interim data. DM1 patients given the experimental therapy in the ongoing ACHIEVE study…

RGX-202, a one-time gene therapy designed to treat Duchenne muscular dystrophy (DMD), has been well tolerated in an ongoing clinical trial, with no serious side effects reported. Interim data from the Phase 1/2 part of the trial, which is sponsored by RGX-202’s developer Regenxbio, also indicate that…