Sarepta Therapeutics, Inc, today announced the U.S. Food and Drug Administration (FDA) has accepted and filed the company's ‘Efficacy Supplement’ to …
This is the latest news about gene therapy development for Duchenne muscular dystrophy. There’s also lots of information and videos explaining these potential treatments in our information pages.
Solid Biosciences Receives FDA Clearance for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
Solid Biosciences Receives FDA Clearance for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 Solid Biosciences Inc., a leading life …
Roche UK issues a statement following Sarepta’s Press release regarding ELEVIDYS FDA decision
Roche UK issues a statement following Sarepta's Press release regarding ELEVIDYS FDA decision Following the press release from Sarepta released on …
Sarepta Therapeutics Announces FDA Approval of ELEVIDYS, the First Gene Therapy to Treat Duchenne Muscular Dystrophy
Sarepta Therapeutics announced U.S. Food and Drug Administration (FDA) accelerated approval of ELEVIDYS (delandistrogene …
Roche UK issues statement to the Duchenne Community following Sarepta’s Update on Regulatory Review of SRP-9001 gene therapy.
Roche UK issues statement to the Duchenne Community following Sarepta's Update on Regulatory Review of SRP-9001 gene therapy. Following the update …
Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001
Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001 Sarepta Therapeutics, today provided the following update on the Biologics …
Sarepta Therapeutics Announces Update on Regulatory Review of SRP-9001Read More
Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular Dystrophy
Sarepta Therapeutics Announces Positive Vote from U.S. FDA Advisory Committee Meeting for SRP-9001 Gene Therapy to Treat Duchenne Muscular …
Sarepta Theraputics Gene Therapy Granted Priority Review by FDA
Sarepta Therapeutics announced that the U.S. FDA has accepted the Company's Biologics License Application for SRP-9001 gene therapy for …
Sarepta Theraputics Gene Therapy Granted Priority Review by FDARead More
Pfizer shares update on Phase 2 DAYLIGHT clinical trial
We are pleased to share an update with you from US based pharmaceutical and biotechnology corporation, Pfizer about their clinical …
Pfizer shares update on Phase 2 DAYLIGHT clinical trialRead More
Nationwide researchers announce restoration of full-length Dystrophin in humans
In Duchenne muscular dystrophy, dystrophin protein is absent or partially functional due to mutations in the dystrophin gene. Multiple therapeutic …
Nationwide researchers announce restoration of full-length Dystrophin in humansRead More
Pfizer to re-start its global Phase 3 Trial of Investigational Gene Therapy for Ambulatory Patients with Duchenne Muscular Dystrophy
Pfizer has announced on 28 April 2022 they have received approvals to re-start the Phase 3 study evaluating their gene therapy for Duchenne muscular …
Gene therapy trial shows ‘statistically significant’ improvements
Sarepta Therapeutics have announced the audited, quality-controlled data reflecting all results from Part 2 of their Study SRP-9001-102 (Study 102). …
Gene therapy trial shows ‘statistically significant’ improvementsRead More