Duchenne Science on Tour 2 Where 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 …
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 …
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 …
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 …
Ignite DMD update
In a press release yesterday, Solid Biosciences gave a short update on their gene therapy trial called Ignite DMD. Following the decision earlier this …
Sarepta issues clinical trials update
In recent press releases, Sarepta Therapeutics has given updates on their ongoing clinical trial programmes. These include exon skipping and gene …
Gene therapy trial update from Solid
Solid Biosciences has announced that the next stage of their clinical trial of a gene therapy has started, following the dose increase announced in …
Solid Biosciences announce preliminary trial results
Solid Biosciences have announced preliminary data from their phase 1/2 IGNITE-DMD clinical trial that is testing the safety and effectiveness of …
Solid Biosciences announce preliminary trial resultsRead More
UNITE-DMD project update – Year 1
Gene therapy for Duchenne muscular dystrophy aims to compensate for the lack of dystrophin by transferring a working version of the dystrophin gene …