PBGENE-DMD aims to deliver sustained muscle function improvement for DMD patients with mutations between exons 45 and 55.

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

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

Muscular Dystrophy Association Marks ALS Awareness Month throughout May with Breakthrough Research, Powerful Stories, and ...

Santhera Pharmaceuticals (SIX: SANN) announces that the Board of Directors of the Italian Medicines Agency, AIFA, has approved the reimbursement of AGAMREE® (vamorolone) for the treatment of patients ...

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

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 ...

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 ...

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.” ...

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