资讯

Fierce Pharma1 天
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 ...
pharmaphorum1 天
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 ...
Second patient death reported with gene therapy for muscular dystrophy
Shares of Sarepta Therapeutics plunged Monday after the biotech drugmaker reported a second death in connection with its gene ...
MedPage Today on MSN1 天
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 ...
Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella ...
Ticker: Second patient death reported with gene therapy for muscular dystrophy; Salmonella poisoning linked to pistachio ...
Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for ...
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
BioWorld1 天
Cure Rare Disease completes pre-IND meeting for limb girdle muscular dystrophy gene therapy ...
Cure Rare Disease has successfully completed a pre-IND meeting with the FDA regarding its investigational gene therapy program for limb-girdle muscular dystrophy type 2I/R9 (LGMDR9).
The American Journal of Managed Care10 天
Advancements & Challenges in Muscular Dystrophy Treatment
Panelists discuss how recent advancements in muscular dystrophy treatment have evolved toward truly disease-modifying ...
PMLive10 天
Duchenne muscular dystrophy – diagnosis, clinical development and global research
Muscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, causing ...
Biotech pauses trial after second patient death linked to gene therapy
Sarepta Therapeutics said a second patient had died after receiving its $3.2 million gene therapy to treat Duchenne muscular ...
MedPage Today on MSN9 天
Second Patient Dies After Gene Therapy for Duchenne Muscular Dystrophy
To date, over 900 Duchenne patients have been treated with delandistrogene moxeparvovec. "The signal for ALF is exceptionally ...