News

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…

A young man with Duchenne muscular dystrophy (DMD) who received the one-time gene therapy Elevidys (delandistrogene moxeparvovec-rokl) has died due to acute liver failure. In a statement, the therapy’s developer, Sarepta Therapeutics, said it is continuing to gather and analyze information about this event, which…

Capricor Therapeutics’ investigational therapy deramiocel, formerly CAP-1002, which is being considered for approval in the U.S., continued to show an ability to preserve upper limb function in boys and men with Duchenne muscular dystrophy (DMD) over the long term. That’s according to analyses of about five years…

The Muscular Dystrophy Association‘s (MDA) annual Clinical & Scientific Conference kicked off over the weekend, bringing together patients, caregivers, researchers, clinicians, academics, advocates, and industry leaders to discuss the latest in science and care related to a range of neuromuscular diseases. This year’s conference will bring together hundreds…

The National Organization for Rare Disorders (NORD) is seeking participants for its survey-based study Living Rare, which aims to better understand the real-world lived experiences of people in the U.S. with rare diseases. Living Rare, the first large-scale study of its kind in the U.S., seeks to capture the…

Asklepios Biopharmaceutical (Askbio) has dosed the first participant in the second group of a Phase 1/2 trial testing AB-1003, an experimental gene therapy for limb-girdle muscular dystrophy (LGMD) type 2I/R9. The Phase 1/2 LION-CS101 trial (NCT05230459) is assessing AB-1003’s safety and tolerability in adults with LGMD2I/R9. Enrollment…