Ifetroban demonstrated significant 5.4% improvement in cardiac functionNASHVILLE, Tenn., March 19, 2025 /PRNewswire/ -- Cumberland Pharma ...

Augustine Therapeutics has raised €78 million ($85 million) in first-round funding for a pipeline headed by a candidate for ...

Patient organisations Muscular Dystrophy UK, Action Duchenne, Duchenne Family Support ... The drug can only be used in people who can’t control symptoms using inhaled corticosteroids and after ...

Cargo was founded by two highly acclaimed cell-therapy researchers. The company raised over $500 million to develop ...

RGX-202, a DMD gene therapy, has been well tolerated in an ongoing clinical trial and improved motor function for DMD boys, per interim data.

A young man with Duchenne MD and treated with Elevidys has died of acute liver failure, Sarepta reports; it's working with ...

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

Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...

The young man died of acute liver injury, a known side effect, Sarepta said in a statement. But the company said the ...

A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...

In 2023, Elevidys received expedited U.S. approval despite concerns from some Food and Drug Administration scientists about its effectiveness in treating Duchenne muscular dystrophy. It’s the ...