Solid begins dosing next-generation gene therapy

We are pleased to share that Solid Biosciences has begun dosing patients in the Phase I/II clinical study of its next-generation gene therapy, SGT-003. The study, INSPIRE DUCHENNE, is ongoing at two sites in the US – Nationwide Children’s Hospital in Columbus, Ohio,...

Sarepta announces gene therapy results

Pharmaceutical company Sarepta has announced results of Part 1 of its Phase 3 Embark gene therapy trial for Duchenne. The trial did not meet its primary endpoint: showing a difference in a change on the North Star Ambulatory Assessment (NSAA) total score – which...

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...

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...
DRF funds CRISPR research to correct Duchenne mutations

DRF funds CRISPR research to correct Duchenne mutations

The Duchenne Research Fund has awarded a £200,000 grant to the team at the Hospital for Sick Children in Toronto, Canada (‘Sick Kids’), who are working on using CRISPR/Cas9 technology to correct duplication mutations for Duchenne. Sick Kids’ incoming President and CEO...