The family of two boys who both have a life-limiting condition are hoping to take a trip to Disneyland Paris later this year.

Patients in Scotland can't get the treatment - which is available in England - despite manufacturers giving it away for free ...

A federal advisory committee that provides guidance on which rare diseases newborns should be screened for has been dissolved ...

Sarepta Therapeutics, Inc. (NASDAQ:SRPT) shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved ...

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular dystrophy treatments was the focus of a recent International Society for ...

The European Medicines Agency asked for three clinical trials to be placed on hold until the exact cause of death of a US ...

Managed Healthcare Executive gives C-suite executives in health plans and provider organizations news and strategies for ...

The Port St. Lucie Police Department fulfilled 10-year-old William Rivero's dream of becoming a police officer by swearing ...

New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing ...

Several companies will head to the FDA seeking approval of new Duchenne muscular dystrophy treatments next year but the death ...