He was diagnosed with Duchenne muscular dystrophy at age 5. "I can remember the day he got diagnosed," said his older sister, ...

Four-year data show preservation of cardiac function, including LVEFSkeletal muscle disease progression continues to slow ...

A North Texas family is sharing a son's journey with Duchenne muscular dystrophy, a rare and progressive muscle-wasting ...

The ouster of the FDA's chief regulator of cell and gene therapies came immediately after a disagreement with her boss over a ...

The Kansas City Chiefs welcomed some special guests to their mandatory minicamp on Tuesday, June 17, and Patrick Mahomes and ...

Sarepta shares face pressure after a second Elevidys-linked death and analyst downgrade over safety concerns and lowered ...

Ovarian cancer remains a significant global health challenge, often diagnosed at an advanced stage due to non-specific ...

Consequently, Truist Securities expects the launch of two new drugs and the development of the pipeline to help offset ...

Robbins LLP reminds stockholders that a class action was filed on behalf of investors who purchased or otherwise ...

During BIO International Convention in Boston USA, the Department of Health – Abu Dhabi (DoH), the regulator of the ...

SynaptixBio announces milestone with selection of candidate drug to treat rare disease H-ABC: Our Bureau, Bengaluru Friday, June 20, 2025, 12:30 Hrs [IST] Oxford-based SynaptixBio ...

The reported dismissal of high-ranking CBER officials Nicole Verdun and Rachael Anatol resurfaced lingering concerns about how gene therapies will be regulated under new FDA leadership.