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…
My husband, Jason, and I are parents to seven children: Lexi, 23; Max, 19; Chance, 17; Rowen, 16; Charlie, 14; Mary, 10; and Callie, 3. This week we’re traveling with five of them: Max, Rowen, and Charlie, who are our three sons with Duchenne muscular dystrophy (DMD), and our…
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…
When I was 13 and my brother, Tim, was 16, we got skis for Christmas. I’m not sure why Mom and Dad decided to get them for us. Jeannette High School, my alma mater, had a ski club, but Tim and I weren’t members. I don’t recall asking for skis.
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…
A close friend of 30 years recently asked what my life was like before I was diagnosed with limb-girdle muscular dystrophy. When I met this man, we were both in college, studying music and playing drums. It was 1991, and I’d been diagnosed with muscular dystrophy for only…
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 (DMD) — and granted it priority review that will speed the agency’s…
Two weeks ago, I stood on stage at the Fullerton Hotel Singapore to receive a Study U.K. Alumni Award for “exceptional efforts in driving inclusion and positive change in society.” The British high commissioner to Singapore handed me the award while my partner, Amanda, and my caregiver, Glenda, cheered me…
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