Five people with oculopharyngeal muscular dystrophy (OPMD) — a type of muscular dystrophy marked by muscle weakness affecting the face and throat — have thus far been treated with the gene therapy candidate BB-301 in an ongoing clinical trial, treatment developer Benitec Biopharma said in a…
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I’ll be straightforward about caregiving: It’s exhausting. I’m the mother of seven children: Lexi, 23, Max, 19, Chance, 17, Rowen, 16, Charlie, 14, Mary, 10, and Callie, 3. I’m also a caregiver to Max, Rowen, and Charlie, who have Duchenne muscular dystrophy (DMD). You are catching me during a challenging…
A Phase 1 clinical trial testing SAT-3247, an oral treatment candidate for Duchenne muscular dystrophy (DMD), is now done enrolling healthy volunteers, its developer Satellos Bioscience announced. “The completion of healthy volunteer enrollment in our Phase 1 trial marks a significant milestone in our mission to develop transformative…
Becoming educated and informed about muscular dystrophy is a good first step. Here you’ll find general information about the disease, including diagnosis, symptoms, and causes.
It helps to know others have been where you are now. Here’s a collection of our columnists’ words of wisdom to help you along on your muscular dystrophy journey.
There is no cure for muscular dystrophy yet, but here you can find more information about current therapeutic approaches, as well as experimental treatments that are in the pipeline.
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