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 ...

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

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 ...

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 ...

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 ...

Amid readouts from the Muscular Dystrophy Association’s annual meeting, the Clinical Trials Arena evaluates five DMD trials ...

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 ...