资讯

Medpage Today on MSN6 小时
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 ...
pharmaphorum8 小时
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 ...
FDA Investigates Patient Deaths After Treatment With Sarepta's Gene Therapy
(Reuters) -The U.S. Food and Drug Administration said on Tuesday that it is investigating reports of two deaths due to acute ...
Fierce Pharma5 小时
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, ...
STAT6 小时
Pharmalittle: We’re reading about FDA probing deaths after Sarepta treatment, a Regeneron ...
The FDA is investigating two deaths in non-ambulatory Duchenne muscular dystrophy patients who received a Sarepta gene ...
Roche Pauses Commercial and Clinical Use of Elevidys After Fatal Liver Failures
The commercial and clinical use of Elevidys was discontinued after two fatal cases of acute liver failure.
Rheumatology Advisor1 天
Second Death Prompts New Safety Initiatives for DMD Gene Therapy Elevidys
Sarepta has issued a safety update regarding Elevidys (delandistrogene moxeparvovec-rokl) following a second reported case of acute liver failure resulting in death.
Managed Healthcare Executive9 天
Sarepta Stops Shipments of Elevidys For Non-Ambulatory DMD Patients
A second patient with Duchenne muscular dystrophy has died from acute liver failure after receiving the gene therapy Elevidys ...
The Week12 天
14-year-old survives acute liver failure, thanks to her mother's grit and timely ...
The only option left for the doctors was liver transplantation and Anakendu's mother came to her rescue by volunteering to ...