Ultragenyx Pharmaceutical and Solid Biosciences have announced a strategic collaboration and license agreement to focus on the development and commercialisation of new gene therapies for Duchenne. The collaboration brings together Solid’s expertise in muscle biology...
Solid Biosciences has issued an update on IGNITE DMD, the Phase I/II clinical study with its microdystrophin gene therapy, SGT-001. The most recent child dosed in the study has experienced a serious adverse event (SAE), and thankfully, he is now improving. He is being...
The Duchenne Research Fund has awarded a grant to Dr Barry Byrne and Dr Manuela Corti’s team at the University of Florida, whose vital work focuses on overcoming immunological challenges to ensure all boys and young men can benefit from gene therapy treatments for...
Solid Biosciences has announced preliminary data from its IGNITE DMD gene therapy clinical trial. The Duchenne Research Fund is proud to have helped seed fund Solid’s gene therapy program together with Duchenne UK and Joining Jack, with a joint investment of $5...
Five years ago we identified gene therapy as a viable treatment for Duchenne. Today we are reflecting on the exciting and inspiring events of the past week in the gene therapy arena which are now bringing hope and cautious optimism to the Duchenne community. We have...
Message from Solid Biosciences: “We’re pleased to share with you that the FDA has lifted the clinical hold on IGNITE DMD, our clinical trial investigating SGT-001 as a potential treatment for Duchenne muscular dystrophy (DMD). SGT-001 is a microdystrophin gene...
We are thrilled that the money raised through the Duchenne Research Fund’s 2013 dinner and donations since then have enabled us to seed fund the incredible Solid GT project, which has today received industry investment (read the press release below). While gene...
We are immensely grateful to Albourne Partners and their supporters for raising more than £155,000 for our projects at their incredible performance of ‘The Importance of Being Lewis’ on 7 October. The event was held to honour the memory of Jacques Joubert...
The Duchenne Research Fund is incredibly proud of Dr Dongsheng Duan – an important Scientific Advisory Board member of Solid GT, a gene therapy programme pioneered by Solid Biosciences and that we helped to fund. Dr Duan’s latest gene therapy work on...
With the funds we raised at our 2013 Gala Dinner we brokered the first partnership between UK Duchenne charities and commercial enterprise to launch Solid GT, an exciting gene therapy project. We approached US-based Solid Biosciences to present promising gene therapy...
