Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant full approval for Amondys 45 (casimersen) and Vyondys 53 (golodirsen), two exon-skipping therapies designed to treat certain people with Duchenne muscular dystrophy (DMD). The company said it met with the FDA to…
Discussion
According to the EveryLife Foundation for Rare Diseases, more than 30 million Americans live with at least one rare disease. Considering that’s nearly 10% of the U.S. population, these diseases don’t seem so rare after all. For decades, the disability rights movement has fought for the rights of…
Muscular Dystrophy News Today is wrapping up coverage of the 2026 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, which ran from March 8-11 in Orlando, Florida, and online. This year, more than 2,400 attendees from 40 countries gathered in person and virtually to discuss recent advances in neuromuscular…
Discussion
The comment comes in many forms: “I don’t know how you do it.” “You have your hands full!” “I can’t imagine what it would be like.” It’s always in response to seeing my large family. I share seven children with my husband, Jason: Lexi, 24; Max, 20; Chance, 18; Rowen,…
Dyne Therapeutics is advancing its investigational exon-skipping therapy zeleciment rostudirsen (z-rostudirsen), formerly known as DYNE-251, toward regulatory approval after trial data showed early signs of benefit across multiple disease measures in boys with Duchenne muscular dystrophy (DMD). The most recent data from the Phase 1/2 DELIVER clinical trial…
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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