Ryuji Morizane, MD, PhD, of the Department of Medicine at Massachusetts General Hospital, is the senior/corresponding author ...

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 ...

Keros Therapeutics (KROS)’ treatment of Duchenne muscular dystrophy was granted FDA orphan designation, according to a post to the agency’s site.

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

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

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

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

University of Manchester scientists have mapped the mutations in the tiny protein chains that cause a subtype of muscular ...

A Champaign second-grader hopes her family's fundraiser can help a kid in the future have a more "normal" childhood, not ...

The Health Resources and Services Administration is requesting public comments on its recommendation to add metachromatic ...

Liquidating its stake in Arrowhead helps Sarepta cover a milestone payment and secure what one analyst described as ...