Some children in Northern Ireland who live with a severe form of muscular dystrophy will be able to access new medication on the health service, BBC News understands. The move from the Belfast Health ...

The U.S. Food and Drug Administration is tightening restrictions on a gene therapy used to treat Duchenne muscular dystrophy after two teenagers died from liver failure linked to the medication. The ...

The family of a boy with a severe degenerative muscle disease has urged their local hospital to prescribe a drug that could help slow down the progress of the condition. Six-year-old Jack was ...

Capricor Therapeutics has received an orphan drug designation for its drug aimed at potentially treating Becker Muscular Dystrophy. The biotechnology company said Tuesday that the Food and Drug ...

A gene therapy for Duchenne muscular dystrophy caused complications to the liver, prompting a review of its use for younger patients. By Christina Jewett The Food and Drug Administration said on ...

The U.S. Food and Drug Administration (FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational gene therapy trials for limb girdle muscular dystrophy following three ...

Jason Mast is a general assignment reporter at STAT focused on the science behind new medicines and the systems and people that decide whether that science ever reaches patients. You can reach Jason ...

Physical activity and rehabilitation are key components of Duchenne muscular dystrophy treatment, despite recent drug therapy advances. "Rehabilitation care and rehabilitation teams with experience ...