News

Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
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 ...
MedPage Today on MSN17h
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 ...
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, ...
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 ...
pharmaphorum19h
FDA probing deaths with DMD gene therapy Elevidys
Elevidys has been given full approval to treat ambulatory patients with DMD, with an accelerated approval in non-ambulatory ...
No Ad Comm ‘not necessarily positive’ for Capricor, says Roth Capital
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 ...
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 ...
Stocktwits on MSN14h
Sarepta Stock Slumps After FDA Opens Probe Into Patient Death, Analyst Sounds Alarm
Brokerage H.C. Wainwright on Wednesday opined that there will be” little intrinsic value” in Sarepta Therapeutics (SRPT) if ...
STAT17h
Pharmalittle: We’re reading about FDA probing deaths after Sarepta treatment, a Regeneron plan for charities, and more
The FDA is investigating two deaths in non-ambulatory Duchenne muscular dystrophy patients who received a Sarepta gene ...
BioSpace2h
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 ...
Asianet Newsable on MSN2h
Precision Bio's DMD Therapy Gets Rare Pediatric Status, But Stock Slides; Retail Crowd Sees ‘Good Risk-Reward’
The FDA designation could make Precision eligible for a valuable Priority Review Voucher, while preclinical data showed ...