News

No Accelerated FDA Approval For Edgewise's Rare Muscular Disease Drug; Shares Fall
Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...
BioSpace18h
Novavax Approval Process a Potential ‘Harbinger’ of Threat to FDA Integrity: Former Regulators
Writing in JAMA, four former government officials warn that the Trump administration’s involvement in delaying the approval ...
Edgewise falls after data for muscular dystrophy therapy
Edgewise Therapeutics (NASDAQ:EWTX) lost ~9% in the morning hours on Thursday after the company posted data from clinical ...
Precision Medicine Online19h
FDA Investigating Fatal Liver Failure Risk With Sarepta's Elevidys
The agency is considering further regulatory action after a second Duchenne patient died from acute liver failure less than two months after receiving the gene therapy.
FDA probes deaths of boys taking Sarepta Duchenne drug
The FDA said yesterday that it is investigating the deaths from liver failure of two non-ambulatory boys with Duchenne ...
Fierce Pharma1d
FDA investigates patient deaths after treatment with Sarepta's Duchenne gene therapy Elevidys
In one of the first major tests of the new FDA leadership’s regulatory philosophy toward gene therapies for rare diseases, ...
MedPage Today1d
FDA Probes Gene Therapy After Two Deaths
Duchenne muscular dystrophy is characterized by a mutation in the DMD gene that leads to a lack of dystrophin and muscle loss ...
Contract Pharma1d
Precision BioSciences Gains FDA Rare Pediatric Disease Designation for PBGENE-DMD
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).
Devdiscourse1d
U.S. Domestic Briefs: Health Challenges, Tech Advancements, and Political Upsets
Recent U.S. domestic news covers the FDA investigating deaths post-gene therapy by Sarepta, discussions at a CDC advisory ...
BioSpace1d
FDA Reviews Sarepta’s Elevidys After Second Patient Death
The FDA is assessing the need for “further regulatory action” on Sarepta's Duchenne muscular dystrophy gene therapy in the ...
FDA investigates patient deaths after treatment with Sarepta's gene therapy
The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute liver ...
Ealing Times2d
Northolt couple tackle peak challenge for their son, 4
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.