MP1032, MetrioPharm’s oral therapy candidate for Duchenne muscular dystrophy (DMD), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). A Phase 2 trial testing MP1032 in DMD patients is expected to start next year. “Currently, DMD cannot be cured, but it can be treated,”…
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Memorial Day weekend has come and gone, and we’re now officially in summer mode at my house. For our family, that means lots of baseball. I have three sons — Max, 17, Rowen, 14, and Charlie, 12 — with Duchenne muscular dystrophy (DMD). But I also have one son,…
The U.S. Food and Drug Administration (FDA) has placed a clinical hold on an application to launch a Phase 1 study of PGN-EDODM1, an experimental medicine by PepGen for people with myotonic dystrophy type 1 (DM1). A clinical hold is an order issued by the FDA to delay…
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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