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 ...

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

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

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

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

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

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 ...

Some studies of Sarepta Therapeutics' muscular dystrophy drug, Elevidys, have been halted by European regulators after a ...

Charley Seckler has found "humor in the madness" of rolling through life with a rare disease. He drops a blog post weekly, ...

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