The U.S. Food and Drug Administration (FDA) is considering whether to conditionally approve zeleciment rostudirsen (z-rostudirsen), an experimental therapy for Duchenne muscular dystrophy (DMD) in patients with mutations that are amenable to exon 51 skipping. Z-rostudirsen developer Dyne Therapeutics announced that the FDA has agreed to review an…
Discussion
As the summer flies by, my thoughts turn to going back to school — reluctantly. As a mother of many, the end of summer is nothing new. I have seven children with my husband, Jason: Lexi, 25; Max, 20; Chance, 19; Rowen, 17; Charlie, 15; Mary, 11; and Callie, 4. Max,…
Discussion
Commendamide, a metabolite produced by beneficial gut bacteria that are depleted in people with Duchenne muscular dystrophy (DMD), protects muscle cells from damage by enhancing antioxidant mechanisms, a study found. “These findings provide new insights into the gut-muscle axis in DMD” and “support further investigation of microbiota-derived metabolites as…
Discussion
I’ve been forced to make lifestyle changes continuously because of the progression of my facioscapulohumeral muscular dystrophy (FSHD) and other health issues. But I’ve regained some independence in the past few weeks after losing some capabilities over the past six months. I have a new sit-to-stand…
Discussion
CureDuchenne, a U.S.-based nonprofit that funds research into new treatments for Duchenne muscular dystrophy (DMD), has launched a national public service announcement campaign to highlight the urgency behind research for people with DMD. The new advocacy campaign, dubbed A Cure Can’t Wait, highlights…
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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