Solid data shows improved motor and lung function

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...
Five-year grant to Dr Michela Guglieri: outcome report

Five-year grant to Dr Michela Guglieri: outcome report

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...

Santhera and ReveraGen share latest vamorolone study results

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...

DRF awards £640,000 for vital psychosocial project

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...

DRF-funded drug shows success in clinical trial

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 An Evening with the DRF

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 receives $3.3m NIH grant for Vamorolone

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...

Solid announces collaboration with Ultragenyx

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...