News

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 ...
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® ...
Fierce Pharma12h
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 Today on MSN13h
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 ...
BioSpace14h
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 ...
pharmaphorum15h
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 ...
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 MSN10h
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 ...
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 ...
STAT13h
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 ...
Contract Pharma13h
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).
Precision BioSciences gets FDA rare disease status for DMD treatment, stock jumps 10%
Precision BioSciences (NASDAQ:DTIL)  announced Wednesday that its experimental therapy PBGENE-DMD has received Rare Pediatric ...