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 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, ...
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 ...
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 ...
Sarepta Therapeutics (NasdaqGS:SRPT) Faces Shipment Suspension Amid Safety Update
Sarepta Therapeutics (NasdaqGS:SRPT) recently provided updates that may have influenced its stock performance. The announcement of a safety update for ELEVIDYS, concerning severe side effects and a ...
pharmaphorum2d
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 ...
Sarepta Viral Vector Gets FDA Platform Technology Designation
Sarepta Therapeutics Inc. (NASDAQ:SRPT) is one of the best mid cap growth stocks to invest in now. Earlier on June 4, Sarepta ...
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 ...
2 Beaten-Down Stocks to Avoid
Not too long ago, Editas Medicine was working on Reni-Cel, an investigational treatment for two rare blood disorders. Although the data from clinical trials for this medicine was promising, Editas ...
Stocktwits on MSN1d
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 ...
MedPage Today on MSN1d
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 ...
GlobalData on MSN4d
Sarepta and Roche halt DMD gene therapy’s use in certain patients after second death
Shares in both companies were down following news of the second death, casting fresh safety concerns over Elevidys.