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Treatment with vamorolone continued to show a good safety profile and sustained efficacy across functional assessments after almost one year in boys with Duchenne muscular dystrophy (DMD), according to 48-week results of the Phase 2b VISION-DMD clinical trial. Data also showed no loss of efficacy in boys who switched…
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Author and advocate Elijah J. Stacy is using what he has learned through adversity in battling Duchenne muscular dystrophy (DMD) to help others overcome their own challenges. DMD is a progressive muscle wasting disease that slowly robs a person’s muscle strength. In Stacy’s case, he lost the ability to…
RGX-202, a one-time experimental gene therapy for the treatment of Duchenne muscular dystrophy (DMD), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA). The designation is given to investigational therapies to treat rare medical conditions or diseases that affect fewer than 200,000 people…
With her three sons with Duchenne MD away at clinic week, columnist Betty Vertin is taking a small break, and learns what free time is like.
Visit the Muscular Dystrophy News Today forums to connect with others in the muscular dystrophy community.
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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