Sarepta Therapeutics has provided additional safety data on its gene therapy Elevidys as a Duchenne muscular dystrophy ...

Parent Project Muscular Dystrophy (PPMD), a nonprofit organization leading the fight to end Duchenne muscular dystrophy (Duchenne), is pleased to announce the expansion of its leadership team with the ...

Managing osteoporosis and bone health in Duchenne muscular dystrophy occurs in a high-risk environment for fractures due to disease-related loss of muscle and movement, adverse effects of ...

Santhera Pharmaceuticals (SIX: SANN) announces the signing of an exclusive agreement with Uniphar to manage the distribution of AGAMREE® (vamorolone) in the United Arab Emirates, the Kingdom of Saudi ...

Dyne Therapeutics Inc. (NASDAQ: DYN) is one of the best small cap stocks with biggest upside potential. On August 4, Dyne ...

Some experts worry that patient groups’ growing influence is leading to the approval of drugs that may not work and could sometimes be dangerous.

The U.S. Food and Drug Administration ( FDA) has placed an immediate clinical hold on Sarepta Therapeutics' investigational ...

The biotech, which cut jobs following the rejection in May of its Barth syndrome therapy, claimed it has addressed the agency ...

Ashfields care home in Rackheath donates £386 to Leon D’Souza for vital home improvements as he battles Duchenne Muscular Dystrophy.

Charlie has Duchenne muscular dystrophy, a degenerative genetic disease that, until recently, has guaranteed death by early adulthood from cardiac or respiratory failure.

Deflazacort, a newly FDA-approved drug for Duchenne muscular dystrophy, has an $89,000-a-year price tag. It has been put on hold because of an uproar over drug prices.

Duchenne Muscular Dystrophy may no longer be a death sentence. Oct. 17, 2012— -- Although he made it to 29 years old with Duchenne muscular dystrophy, Jason Williams was not expected to ...