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 ...
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 Today on MSN16h
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 ...
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 ...
BioSpace17h
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 ...
pharmaphorum18h
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 ...
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
FDA has indicated that an Advisory Committee meeting is not required at this timeIn-person late-cycle review meeting ...
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® ...
STAT16h
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 ...
Stocktwits on MSN1d
Capricor Therapeutics Says FDA May Not Hold Advisory Committee Meeting For DMD Therapy Approval: Stock Soars But Analyst Issues Warning
Capricor Therapeutics (CAPR) said on Tuesday that the U.S. Food and Drug Administration is not eyeing an advisory committee ...