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 ...

Investigational deramiocel is back under FDA review again ...

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

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

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

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 ...

Muscle tissue damage appears when muscular dystrophy is induced in a mouse model (middle). But when researchers block the function of two genes that drive unwanted mitochondrial pore formation (right) ...

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

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.” ...