Dyne Therapeutics’ investigational therapy DYNE-101 has been granted an orphan drug designation by the European Medicines Agency (EMA) for myotonic dystrophy type 1 (DM1). The designation is meant to support the development of potential therapies for rare, life-threatening, or chronically debilitating diseases. The benefits include reduced fees, clinical…
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He’s getting weaker. That’s the thought that keeps me up at night. Rowen, 14, the second of my three sons with Duchenne muscular dystrophy (DMD), is declining. Rowen has always been the weakest of my three boys with DMD, and the disease has progressed faster for him than for…
The U.S. Food and Drug Administration (FDA) has pushed back its decision on SRP-9001 (delandistrogene moxeparvovec), an experimental gene therapy for Duchenne muscular dystrophy (DMD). A decision is now expected by June 22, according to a press release. If the agency decides to approve SRP-9001, it’s expected to be…
Columnist Robin Stemple debates whether he should give physical therapy another try as someone with facioscapulohumeral muscular dystrophy.
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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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