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...
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...
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...
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...
We are pleased to share the announcement from Santhera Pharmaceuticals and ReveraGen BioPharma of new topline results after completion of the VISION-DMD study at week 48. VISION-DMD was a pivotal double-blind Phase 2b study designed to demonstrate efficacy and safety...
The Duchenne Research Fund is delighted to announce that we are awarding a £640,000 grant to fund vital psychosocial care and research as part of Duchenne UK’s DMD Care UK programme. We are so pleased to partner with Duchenne UK on this important project. The...
We are pleased to share that Vamorolone, an alternative medicine to corticosteroids, has shown positive results in a Phase 2 Study. Vamorolone was developed as a treatment for Duchenne muscular dystrophy, to offer the benefits of steroids, but without some of the side...
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...
ReveraGen BioPharma has received a $3.3 million grant from the NIH – the US National Institutes of Health – for their ongoing work on the steroid alternative treatment Vamorolone for Duchenne muscular dystrophy. The NIH Commercialization Readiness Pilot...
