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...

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...

Gene therapy for Duchenne: latest news

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...