News

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 ...
Pharmabiz7h
Edgewise Therapeutics announces positive results on sevasemten programme for Becker and Duchenne muscular dystrophies
Currently, there are no approved therapies on the market to treat Becker. Duchenne, a severe degenerative muscle disorder, is the most common type of muscular dystrophy with a median life expectancy ...
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 ...
BioSpace11h
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 Pharma2d
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, ...
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 ...
STAT11d
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 ...
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 ...
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® ...
Game Center
Every year, thousands of infants in America are diagnosed with devastating genetic diseases through newborn screenings. The ...
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 ...
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 ...