From STAT’s Adam Feuerstein: The FDA informed Capricor Therapeutics that a meeting of outside experts will be convened to ...

It’s a devastating diagnosis that changes a child’s life — and their family’s — forever. Duchenne Muscular Dystrophy, or DMD, is a rare and fatal genetic disease that primarily affects boys, slowly ...

While right-wing populism surges in some of the world’s leading democracies, Chile’s leftist government is rushing in the ...

A new study led by researchers at the Hospital for Special Surgery (HSS) suggests that currently available therapies may help ...

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 ...

Michael Rankin, 12, has Duchenne Muscular Dystrophy, a condition which causes progressive muscle weakness. He has been using a wheelchair for five years now. He can still walk but only for very ...

Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing them to weaken and deteriorate over time. While muscular dystrophies are ...

This dystrophin transcript, theoretically, would exist in both muscle and the central nervous system, resulting in Duchenne muscular dystrophy in the former and neuronal dysfunction in the latter ...

“There are no disease-modifying treatments approved for patients with any subtype of limb-girdle muscular dystrophy, and the unmet medical need is significant. Following feedback from U.S ...

SAN DIEGO — Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory boys with Duchenne muscular dystrophy, interim data show.

Despite being known for more than 150 years, Duchenne muscular dystrophy (DMD) remains an untreatable disease affecting approximately 1 of every 3,500-5,000 males. Muscles in patients express no or ...

The product is the generic equivalent of Emflaza tablets, and is indicated for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 5 years and older. Jaythari will be manufactured at ...