On Rare Disease Day, watch Eli Crossley, son of our friend and Duchenne UK co-founder Emily, as he talks about – and demonstrates – the amazing technological developments that are being trialled for children with Duchenne muscular dystrophy. The ‘smart...
DMD Care UK’s new cardiac care recommendations have been published in the BMJ Open Heart journal. All Duchenne parents and caregivers are being urged to read the new guidance and share with their networks, as protecting the heart early is crucial to preventing...
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...
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...
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...
Dr Ronald Cohn, who leads the Duchenne Research Fund’s Scientific Advisory Board, has become the first ever researcher to successfully correct one of the mutations that causes Duchenne muscular dystrophy, working on cells in his laboratory at the Centre for...
DRF-funded research was published this week in Cell, one of the world’s most prestigious scientific journals, analysing why two golden retrievers showed no severe symptoms of Duchenne muscular dystrophy and unexpectedly lived a normal lifespan despite being born...
