Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today unveiled positive ...

Capricor Therapeutics updates on Deramiocel BLA for duchenne muscular dystrophy: San Diego Thursday, June 26, 2025, 15:30 Hrs [IST] Capricor Therapeutics, a biotechnology company ...

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

The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation to PBGENE-DMD, an experimental ...

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 ...

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

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).

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