I know what you’re thinking: another awareness day. One more health observance to briefly acknowledge for 24 hours, then mindlessly scroll past once it’s over. But for ultra-rare neuromuscular conditions, awareness is everything. Disease-specific awareness days actually matter. They create exposure, support, belonging, hope, and collaboration that can have a…
Discussion
Dyne Therapeutics expects to report top-line data in early 2027 from its Phase 1/2 ACHIEVE trial testing zeleciment basivarsen (z-basivarsen), an experimental treatment for myotonic dystrophy type 1 (DM1). The company announced that enrollment is now complete for this key group of patients. If the results are positive,…
Discussion
Adults with muscular dystrophy (MD) in the U.S. report widespread gaps in sexual and reproductive healthcare, according to surveys and interviews. Such gaps included a lack of private clinical time, provider discomfort, and unaddressed concerns about fertility and relationships. At the same time, participants indicated a strong desire for…
Discussion
Here are some facts about me: I am an introvert, I am quiet, and my strongest voice is the written word. I grew up in the foster care system and experienced childhood trauma that makes it hard for me to know how to be around people. I am a mom…
Discussion
No treatments are currently approved and available for limb-girdle muscular dystrophy type 2i (LGMD2i) in the U.S., but that may change later this year. The U.S. Food and Drug Administration (FDA) has accepted an application from Bridgebio Pharma seeking approval for its experimental oral therapy BBP-418. The…
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.
Get regular updates to your inbox.
