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...
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...
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,...
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...
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...
The Duchenne Research Fund has awarded a grant to Dr Barry Byrne and Dr Manuela Corti’s team at the University of Florida, whose vital work focuses on overcoming immunological challenges to ensure all boys and young men can benefit from gene therapy treatments for...
