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 ...

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

LUBBOCK, Texas (KCBD) - A Lubbock woman is turning her disability into a super power. Steffni Randale was diagnosed with ...

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

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

The first gene therapy for children with Duchenne muscular dystrophy has been approved by the U.S. Food and Drug Administration. The therapy can be used in 4- and 5-year-olds with the degenerative ...

El Pasoan Angelina Olivera has already buried two brothers after they were diagnosed with Duchenne muscular dystrophy.Now, ...

Delandistrogene moxeparvovec, a gene therapy for Duchenne muscular dystrophy (DMD), showed high dystrophin expression and a favorable safety profile in young patients. Delandistrogene moxeparvovec ...

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 ...

Santa Cruz community raises $97,000 for adaptive van for boy with Duchenne Muscular Dystrophy ...

Tributes poured in for Gilbert Gottfried on Tuesday after news broke that he died in Manhattan. The 67-year-old comedian’s family shared on Twitter that he passed away “after a long illness.” ...