资讯

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® ...
BioSpace20 小时
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 ...
Fierce Pharma18 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, ...
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 ...
pharmaphorum21 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 ...
MedPage Today on MSN1 天
FDA Probes Gene Therapy After Two Deaths
The FDA is investigating the deaths of two boys with Duchenne muscular dystrophy who died soon after being treated with ...
Press Enterprise Online4 天
Gene therapy for muscular dystrophy sees a second death
Shares of Sarepta Therapeutics plunged after the biotech drugmaker reported a second death in connection with its gene therapy for muscular dystrophy.
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 ...
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular ...
FDA has indicated that an Advisory Committee meeting is not required at this timeIn-person late-cycle review meeting ...
Asianet Newsable on MSN3 小时
Precision Bio's DMD Therapy Gets Rare Pediatric Status, But Stock Slides; Retail Crowd Sees ...
The FDA designation could make Precision eligible for a valuable Priority Review Voucher, while preclinical data showed ...
Muscular Dystrophy News1 天
FDA advisory committee meeting for deramiocel in DMD set for July
The U.S. Food and Drug Administration is due to review data on deramiocel, a cell therapy up for approval for heart disease ...
pharmaphorum3 天
Sarepta files Duchenne muscular dystrophy gene therapy with FDA
Sarepta Therapeutics has followed through on its promise to file for accelerated approval of its gene therapy SRP-9001 for Duchenne muscular dystrophy (DMD), as it aims for a launch in the middle ...