Suneel Ram is a spiritual seeker, sings in a band and was the subject of a comic novel two years ago about living with Duchenne muscular dystrophy, a rare genetic ...

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

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

Shares of Sarepta Therapeutics, Inc. (SRPT) traded over 2% lower in afternoon trade on Friday after the company said it has ...

In response to a request from the European Medicines Agency (EMA) following the death of a 16-year-old patient in the U.S., ...

Wave Life Sciences’ experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, setting the ...

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

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 ...

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

An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.

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

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