News

North Texas family raising awareness of muscular dystrophy struggles
He was diagnosed with Duchenne muscular dystrophy at age 5. "I can remember the day he got diagnosed," said his older sister, ...
Capricor Therapeutics Provides Regulatory Update on Deramiocel BLA for Duchenne Muscular Dystrophy
FDA has indicated that an Advisory Committee meeting is not required at this timeIn-person late-cycle review meeting ...
Precision BioSciences Receives FDA Rare Pediatric Disease Designation for PBGENE-DMD for the Treatment of Duchenne Muscular Dystrophy
Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...
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 ...
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 ...
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 ...
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, ...
BioSpace2d
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 ...
Pharmabiz1d
Capricor Therapeutics updates on Deramiocel BLA for duchenne muscular dystrophy
Capricor Therapeutics updates on Deramiocel BLA for duchenne muscular dystrophy: San Diego Thursday, June 26, 2025, 15:30 Hrs [IST] Capricor Therapeutics, a biotechnology company ...
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 ...
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 ...
STAT6d
Disagreements over Duchenne therapy, management style may have led to ouster of key FDA official
The ouster of the FDA's chief regulator of cell and gene therapies came immediately after a disagreement with her boss over a ...