SOLVE FSHD, a venture philanthropy organization dedicated to accelerating treatments for facioscapulohumeral muscular ...

During ALS Awareness Month this May, the Muscular Dystrophy Association (MDA) is highlighting unprecedented scientific progress, inspiring personal stories, legislative advocacy, and national ...

Orphan drug designation from the European Commission underscores Solid’s commitment to advance SGT-003 through a global development effort for ...

“When they diagnosed me they said the muscles around my heart would give out and they gave me [a life expectancy of] 7. Now I ...

Jon Anning, a Slippery Rock University professor, has done extensive research on effects and treatment for Duchenne muscular ...

Your muscles need continuous maintenance to stay strong and healthy. Duchenne muscular dystrophy (DMD) happens when there is a change in a person's genetic instructions that affects the production of ...

Burn Boot Camp 'Be Their Muscle' Campaign to benefit Muscular Dystrophy Association throughout April 2026 Burn Boot Camp celebrates 10 years of partnership with the Muscular Dystrophy Association, ...

Santhera Pharmaceuticals (SIX:SANN, OTC:SPHDF, FRA:S3F0), the Swiss rare disease specialist, has received a positive opinion ...

The EMA has backed broader pediatric use of Agamree and Crysvita, lowering age thresholds for rare genetic diseases affecting muscle and bone development.

Emma Ciafaloni, MD, FAAN, dives into the latest developments in gene therapy for muscular dystrophies, focusing on Duchenne muscular dystrophy, and discusses challenges, genetic causes, and the ...

Duchenne muscular dystrophy (DMD) is a genetic condition caused by a change, or mutation, in the DMD gene. This gene normally tells the body how to make dystrophin, a protein that helps protect ...

We hypothesize that Duchenne muscular dystrophy and autism spectrum disorder/pervasive developmental disorder co-occur with a greater than random frequency. In this study, we set out to reject the ...