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

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 ...

Sarepta stock plunged Thursday after European officials put all studies of the company's gene therapy, Elevidys, on hold.

On April 25, Joe Jackson and a dedicated crew of men including Jeremy Leever and Shaun Olson will embark on an extraordinary nonstop, overnight rowing journey covering 157 miles of the Rogue River to ...

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

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

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

Participants in the FORWARD-53 trial had clinically significant improvements in time-to-rise, among other metrics.

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

Duchenne muscular dystrophy (DMD ... given the effects the disease has on the muscles and bones, which can be worsened by the person not getting adequate nutrition. Read on to learn more about ...

The Cambridge-based biotech's experimental treatment for Duchenne muscular dystrophy hit the mark in a mid-stage study, ...