Patient advocate Donavon Decker has been given the 2025 Â鶹Éç 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 (Â鶹Éç)’s annual…
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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 (Â鶹Éç), 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 (Â鶹Éç) 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 (Â鶹Éç) over the long term. That’s according to analyses of about five years…
The Muscular Dystrophy Association‘s (Â鶹Éç) 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…
The U.S. Food and Drug Administration (FDA) has accepted Capricor Therapeutics’ application seeking approval of deramiocel — a cell therapy developed by the biotech company to treat heart muscle disease in people with Duchenne muscular dystrophy (Â鶹Éç) — and granted it priority review that will speed the agency’s…
Note: This story has been updated March 6, 2025, to correct a quote from Mindy Henderson, Â鶹Éç vice president of disability outreach and empowerment. The Muscular Dystrophy Association (Â鶹Éç) is once again hosting its annual gathering, the Â鶹Éç Clinical & Scientific Conference. The 2025 event will take place…
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