News

BioSpace3h
Novavax Approval Process a Potential ‘Harbinger’ of Threat to FDA Integrity: Former Regulators
Writing in JAMA, four former governmental officials warn that the Trump administration’s involvement in delaying the approval ...
Stocktwits on MSN3h
Precision Bio's DMD Therapy Gets Rare Pediatric Status, But Stock Slides; Retail Crowd Sees ‘Good Risk-Reward’
Precision BioSciences drew heightened retail investor chatter on Wednesday after the FDA awarded Rare Pediatric Disease ...
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 ...
Did The FDA Make A Mistake? Sarepta's Elevidys Approval Under Scrutiny
FDA is investigating Sarepta's Elevidys after two deaths from liver failure in non-ambulatory DMD patients, raising safety ...
Asianet Newsable on MSN13h
Sarepta Stock Slumps After FDA Opens Probe Into Patient Death, Analyst Sounds Alarm
H.C. Wainwright reiterated a ‘Sell’ rating on Sarepta on Wednesday with a $10 price target. The firm views the FDA as a ...
Fierce Pharma16h
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 Today17h
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 Pharma17h
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).
Devdiscourse18h
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 ...
BioSpace18h
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 ...
Two Children Dead After Gene Therapy: Florida Attorney Raises Alarms About FDA Oversight
A second child has died after receiving Elevidys, an experimental gene therapy for Duchenne muscular dystrophy—raising serious concerns about regulatory shortcuts, patient safety, and the urgent need ...