It has been a busy time in the Duchenne arena. In the US, the FDA has granted accelerated approval for the gene therapy treatment SRP-9001. This is the first time a gene therapy for Duchenne has been approved anywhere in the world, and represents a very exciting step...
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...
Solid Biosciences has announced its entry into a definitive merger agreement to acquire AavantiBio, to create a genetic medicines company focused on neuromuscular and cardiac rare diseases, maintaining a strong focus on Duchenne. In a letter to the Duchenne community,...
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...
Watch the highlights of Gavriel Rosenfeld in conversation with Dr Ronni Cohn at our virtual event: An Evening with the Duchenne Research Fund. More than 400 people tuned in to hear Gavriel talk about living with Duchenne, mental health, juggling school and hospital...
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...
