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 trials."Following …
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
In just 7 weeks, we’ll be on the road again
Duchenne Science on Tour 2Where we'll be helping our families to make informed choices around really complex things like gene therapy and exon …
Gene therapy experts announced
Have we got an exciting line up of speakers and sessions for you!We will be announcing more details over the coming days and weeks.But for now, to …
Latest results from Solid gene therapy trial
Solid Biosciences have today released an update on their clinical trial of a gene therapy for Duchenne. The IGNITE DMD trial is an early stage …
Visit to Royal Holloway
Yesterday, we were delighted to be a part of the Royal Holloway University of London's Rare Disease Day event.Neil and Helen were inspiring the next …
Update on UNITE-DMD project
The UNITE-DMD project, which we are co-funding alongside Muscular Dystrophy UK and AFM-Téléthon brings together several strands of research to …
Duchenne Science events back on the road in 2020
We've welcomed over 100 people to our Science on Tour events throughout 2019, these empowering and invaluable sessions have been a huge success and we …
Solid gene therapy trial placed on hold
Solid BioSciences has announced that the IGNITE DMD trial - a Phase I/II clinical trial of the company’s SGT-001 gene therapy - has been placed …
