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...
Gavriel Rosenfeld talks to Dr Ronni Cohn about living with Duchenne

Gavriel Rosenfeld talks to Dr Ronni Cohn about living with Duchenne

On Wednesday 17th March the Duchenne Research Fund hosted a virtual evening to share updates with supporters about research funded by the charity. Our co-founders Kerry & Doron Rosenfeld welcomed hundreds of viewers from across five continents who came together...

DRF funds Duchenne clinic at Great Ormond Street

We are pleased to share that we have awarded a grant of £35,000 to Great Ormond Street Hospital to establish a dedicated clinic for children who are non-ambulant (no longer able to walk). The grant supports the post of Senior Physiotherapist Nicola Burnett (pictured)...

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...

Two-year Vamorolone study completed

Santhera Pharmaceuticals has announced that partner ReveraGen Biopharma has completed a long-term, open-label extension study of 24 months duration with Vamorolone in patients with Duchenne muscular dystrophy. Including 6 months treatment in the preceding study,...

Update on Ignite DMD from Solid Biosciences

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...
DRF-funded research featured on ITV’s Little Heroes

DRF-funded research featured on ITV’s Little Heroes

We are so proud that the family of our founders, Kerry and Doron Rosenfeld, starred in the first episode of season 2 of Paul O’Grady’s Little Heroes on ITV this week, highlighting the ground-breaking activity monitoring study that the Duchenne Research...