News

Edgewise falls after data for muscular dystrophy therapy
Edgewise Therapeutics (NASDAQ:EWTX) lost ~9% in the morning hours on Thursday after the company posted data from clinical ...
BioSpace1d
Edgewise Takes a Hit as FDA Blocks Accelerated Path for Becker Muscular Dystrophy Drug
The FDA found that data from a single Phase II study were “insufficient” to justify an accelerated approval review for ...
Fierce Pharma3d
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 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 ...
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 ...
STAT12d
After second Sarepta death, Duchenne muscular dystrophy community is racked by recrimination and worry
Another gene therapy death of a Duchenne patient has some parents and doctors criticizing the FDA's actions, even as other ...
Edgewise Therapeutics Reports Positive Results On Sevasemten Program For Becker And Duchenne Muscular Dystrophies
The MESA data demonstrated sustained disease stabilization, reinforcing prior ARCH and CANYON findings. Importantly, CANYON ...
Second patient death reported with gene therapy for muscular dystrophy
Shares of Sarepta Therapeutics have plunged again after the drugmaker reported a second death in connection with its gene ...
Game Center
Every year, thousands of infants in America are diagnosed with devastating genetic diseases through newborn screenings. The ...
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® ...
MedPage Today on MSN12d
Second Patient Dies After Gene Therapy for Duchenne Muscular Dystrophy
Duchenne muscular dystrophy patient died after being treated with the gene therapy delandistrogene moxeparvovec (Elevidys), ...
Sarepta reports second patient death after treatment with Duchenne gene therapy
Cambridge-based Sarepta Therapeutics said it was halting shipments of its Duchenne muscular dystrophy gene therapy for ...