Patient-led nonprofit The Speak Foundation has announced the launch of the LGMD Centers of Excellence, a novel alliance of…
Articles by Marisa Wexler, MS
Upsher-Smith Laboratories has launched two new websites to support people with Duchenne muscular dystrophy (DMD) who are taking…
A new collaboration aims to improve the design of clinical trials testing potential treatments for facioscapulohumeral muscular dystrophy (FSHD).
The U.S. Food and Drug Administration (FDA) has given Mesoblast the go-ahead to launch a clinical trial testing its…
Boys with Duchenne muscular dystrophy (DMD) may be able to regain lost height and overcome growth stunting by switching…
Bridgebio Pharma has submitted an application asking the U.S. Food and Drug Administration (FDA) to approve its experimental oral…
Sarepta Therapeutics plans to ask the U.S. Food and Drug Administration (FDA) to grant full approval for Amondys…
PGN-EDODM1, Pepgen’s experimental therapy for myotonic dystrophy type 1…
Becker muscular dystrophy (BMD) patients who received the experimental treatment sevasemten in clinical trials had stable motor function…
Four people with oculopharyngeal muscular dystrophy (OPMD), a disease type marked by swallowing difficulties, experienced long-lasting improvements in their…