Writing in JAMA, four former governmental officials warn that the Trump administration’s involvement in delaying the approval ...

Precision BioSciences drew heightened retail investor chatter on Wednesday after the FDA awarded Rare Pediatric Disease ...

The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...

FDA is investigating Sarepta's Elevidys after two deaths from liver failure in non-ambulatory DMD patients, raising safety ...

Osteoarthritis drug developer Paradigm says its purchase of an oral drug candidate won't distract it from its key phase III knee trial.

In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...

Duchenne muscular dystrophy is characterized by a mutation in the DMD gene that leads to a lack of dystrophin and muscle loss ...

Precision BioSciences, Inc. received a Rare Pediatric Disease Designation from the U.S. Food and Drug Administration (FDA) for PBGENE-DMD to treat Duchenne muscular dystrophy (DMD).

Recent U.S. domestic news covers the FDA investigating deaths post-gene therapy by Sarepta, discussions at a CDC advisory ...

The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...

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

A NORTHOLT couple are taking on the Pedal Paddle Peak challenge in the Lake District on Saturday, July 5. It involves a 34km cycle, 3km canoe paddle and 17km hike to the peak of Helvellyn.