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…
Discussion
During a recent facioscapulohumeral muscular dystrophy (FSHD) wellness group meeting via Zoom, a friend of mine shared a poem by May Sarton that prompted the question: “Do I think of my body as an old friend or as an enemy?” I realized that my attitude toward my body has…
Discussion
Seeking to build on the positive results seen among boys with Duchenne muscular dystrophy (DMD) treated with its experimental exon-skipping therapy zeleciment rostudirsen — z-rostudirsen, formerly known as DYNE-251 — in an early clinical trial, Dyne Therapeutics has launched a global confirmatory study, dubbed FORZETTO. This new Phase 3…
Discussion
Having a rare disease is not an easy life. I have limb-girdle muscular dystrophy, so in addition to the physical decline of my body, I also need to be hyperaware of my mental health. I don’t think humans were hardwired to adjust to life on their own with a…
Discussion
An experimental gene therapy called ATA-200 has shown promising safety and efficacy results in an early clinical trial for children with limb-girdle muscular dystrophy type R5 (LGMDR5). Long-term data from the first two treated patients suggest the therapy is working as expected, with no serious side effects, according 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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