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

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 ...

H.C. Wainwright reiterated a ‘Sell’ rating on Sarepta on Wednesday with a $10 price target. The firm views the FDA as a ...

Roth Capital analyst Boobalan Pachaiyappan tells investors in a research note that the FDA’s intention not to require an Ad Comm “at this time” for deramiocel for the treatment of cardiomyopathy ...

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

The FDA is investigating two deaths in non-ambulatory Duchenne muscular dystrophy patients who received a Sarepta gene ...

Precision BioSciences (NASDAQ:DTIL)  announced Wednesday that its experimental therapy PBGENE-DMD has received Rare Pediatric ...

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).

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

Elevidys has been given full approval to treat ambulatory patients with DMD, with an accelerated approval in non-ambulatory ...