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...
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...
We are delighted to share the news that Dr Ronald Cohn, the head of the DRF’s Scientific Advisory Board, has been appointed President and CEO of the Hospital for Sick Children in Toronto, Canada (known as ‘Sick Kids’), effective 1 May 2019. Dr Cohn...
The Duchenne Research Fund has awarded a £200,000 grant to the team at the Hospital for Sick Children in Toronto, Canada (‘Sick Kids’), who are working on using CRISPR/Cas9 technology to correct duplication mutations for Duchenne. Sick Kids’ incoming President and CEO...
We are pleased to share an update from ReveraGen about its Vamorolone clinical trial. Vamorolone is an alternative treatment to existing steroids that aims to maximise the benefits while reducing the side effects. In 2014, The Duchenne Research Fund helped fund the...
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...
The Duchenne Research Fund was delighted to participate in the Duchenne Hub information day held on 18 September 2017 at Alder Hey Children’s Hospital in Liverpool. The event was hosted by Duchenne UK, organised in conjunction with Hub manager Emma Heslop and the...
