We are pleased to share that Solid Biosciences has completed its acquisition of gene therapy company AavantiBio, to create a genetic medicine company focused on neuromuscular and cardiac rare disease, with a continued, strong focus on Duchenne. At this pivotal moment...
Dedicated specialist support for Duchenne muscular dystrophy (DMD) patients and families’ mental health and wellbeing will soon be more readily available, as part of the DMD Care UK project. DMD Care UK is a nationwide initiative aiming to ensure that every person...
We are pleased to share Solid Biosciences’ latest update on the IGNITE DMD gene therapy clinical trial. Data after two years suggests that patients’ motor function and lung function are improved compared to the declines that would be expected without...
We are delighted to report the outcomes of our grant for a Clinical Trials Lectureship. The grant, which was supported by a consortium of seven UK charities, saw the Duchenne Research Fund joined by Action Duchenne, Alex’s Wish, Duchenne Now, Duchenne UK, Harrison’s...
On Wednesday 17th March the Duchenne Research Fund hosted a virtual evening to share updates with supporters about research funded by the charity. Our co-founders Kerry & Doron Rosenfeld welcomed hundreds of viewers from across five continents who came together...
We are delighted to announce that Duchenne Research Fund has awarded £40,000 to the newly launched DMD Care UK, partnering with the project’s founders Duchenne UK and Joining Jack. Together our three charities will invest £130,000 in the nationwide initiative...
Santhera Pharmaceuticals has announced that partner ReveraGen Biopharma has completed a long-term, open-label extension study of 24 months duration with Vamorolone in patients with Duchenne muscular dystrophy. Including 6 months treatment in the preceding study,...
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...
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...
