Shortly after being diagnosed with muscular dystrophy in 1985, I returned to the hospital for a checkup and to review my treatment plan. I thought the doctors might tell me to eat an apple, take some aspirin, and rest up. I was 12 years old, so what did I…
Discussion
The University of Alabama at Birmingham (UAB) has launched an expanded access program to make the experimental exon-skipping therapy delpacibart zotadirsen (del-zota) available to qualifying patients with Duchenne muscular dystrophy (DMD). Del-zota, which is being developed by Avidity Biosciences, is specifically designed to treat DMD in people…
Discussion
An advisory committee to the U.S. Food and Drug Administration (FDA) has voted that available evidence does not support the effectiveness of the experimental cell therapy deramiocel for treating heart muscle disease in people with Duchenne muscular dystrophy (DMD). In a company press release, Capricor Therapeutics,…
Discussion
The first thing I tell others about myself is that my husband and I share seven children: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Being a mom is literally my favorite thing about myself. Caring for my family is my main purpose…
Discussion
The next Clinical & Scientific Conference hosted by the Muscular Dystrophy Association (MDA) will take place March 21-24, 2027, in Orlando, Florida, and registration for the signature annual event — dubbed by the MDA “the leading international meeting dedicated to neuromuscular diseases” — is now open. The conference, to…
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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