The U.S. Food and Drug Administration (FDA) is extending its review of deramiocel, an experimental cell therapy for Duchenne muscular dystrophy (DMD) as it reviews additional data. Capricor Therapeutics, the company developing deramiocel, said it amended its application seeking FDA approval to include new long-term data from…
Discussion
In the spring of 1986, my doctor told my parents and me that one of the standard prescribed treatments for muscular dystrophy was splints, which I would wear on my legs to keep my heel cords loose and limber. The doctors wanted my ankles to reach a 90-degree bend,…
Discussion
The week before school started was pure chaos. We had only been home from vacation long enough to unpack and start the laundry before we had to go to open houses on Monday at two of the kids’ schools, and then close on our new house. If that…
Discussion
Epicrispr Biotechnologies has raised $90 million in financing to support late-stage clinical testing of EPI-321, the company’s epigenetic treatment candidate for facioscapulohumeral muscular dystrophy (FSHD). “This financing marks a pivotal milestone for Epicrispr as we advance EPI-321 and the next generation of programmable epigenetic medicines,” Amber Salzman, PhD,…
Discussion
Last year, our family’s wheelchair-accessible van broke down due to a fault in its hydraulic ramp. From March to May 2025, my caregiver and I had no choice but to take the bus to my office. It was the first time I’d used public transportation in a decade. Back then,…
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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