Spinal muscular atrophy (SMA) is a genetic neuromuscular disease affecting specialized nerve cells that control voluntary ...

Shares of several biotech firms fell in premarket trading Monday after reports that ousted U.S. Food and Drug Administration vaccine regulator Vinay Prasad will return to the agency.

In the wake of multiple patient deaths from liver injuries related to Sarepta Therapeutics’ AAV gene therapy platform, some ...

An extreme athlete has become only the 63rd person to complete the Enduroman Arch 2 Arc challenge, raising more than £21,000 for a muscular dystrophy charity. Sam Weale, from Monmouth, swam, ran and ...

Sarepta Therapeutics said Friday that the deaths of two patients taking its marketed gene therapy Elevidys® (delandistrogene ...

The Duchenne Muscular Dystrophy drug is available elsewhere in the UK but the Belfast Trust has opted against the early ...

The Wigan 10k is set to reach a huge fund-raising milestone when runners toe the start line next month. For it is on course for the total amount of money raised since its launch in 2012 to reach a ...