Muscular Dystrophy News

1 week ago | musculardystrophynews.com | Marisa Wexler |Patrick Moeschen |Lindsey Shapiro

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 already in clinical testing for other types of LGMD.

2 weeks ago | musculardystrophynews.com | Marisa Wexler |Betty Vertin

I recently had the opportunity to attend a Singaporean theater production titled “Supervision,” thanks to the SingHealth Patient Advocacy Network (SPAN). SPAN is a collective of patients and caregivers who provide feedback to improve healthcare here in Singapore. I’ve been part of it since 2022. The invitation to the play was a thank-you for volunteering with several healthcare projects, including reviewing fact sheets on chronic illnesses for the Health Ministry’s Agency for Care Effectiveness.

2 weeks ago | musculardystrophynews.com | Andrea Lobo

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 received the gene therapy died due to acute liver failure.

3 weeks ago | musculardystrophynews.com | Marisa Wexler |Betty Vertin |Margarida Maia

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 were noted. Data also indicate that KER-065 is working as designed, according to developer Keros Therapeutics.

1 month ago | musculardystrophynews.com | Marisa Wexler |Andrea Lobo |Lindsey Shapiro

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 meeting, recently held in Dallas and virtually.