The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...

In a step toward treating mitochondrial diseases, researchers in the Netherlands have successfully edited harmful mutations ...

Sarepta has issued a safety update regarding Elevidys (delandistrogene moxeparvovec-rokl) following a second reported case of acute liver failure resulting in death.

Nobel Prize-winning scientist Dr. Drew Weissman has co-founded a new biotech company in Ambler with the mission of reversing ...