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Muscular Dystrophy News7 小时
Precision’s gene-editing therapy gets FDA’s rare pediatric disease status
The FDA has granted rare pediatric disease designation to PBGENE-DMD, an experimental gene-editing therapy for Duchenne ...
BioSpace1 天
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 ...
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® ...
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 ...
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 ...
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, ...
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).
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 ...
No Accelerated FDA Approval For Edgewise's Rare Muscular Disease Drug; Shares Fall
Edgewise reports sustained functional stability in Becker patients and selects Phase 3 dose for Duchenne after positive Phase ...
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.
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 ...
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 ...