Combating immunity

Combating immunity to AAV Ensuring as many patients as possible can benefit from gene therapy 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...

CRISPR

CRISPR for Duchenne Genome editing technology to correct the mutated dystrophin gene 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...

Vamorolone

Vamorolone Alternative to existing steroids for Duchenne patients The only current standard of care for boys with Duchenne is early treatment with long-term steroids, which gives some protection to muscles but with significant side effects. In July 2014, alongside...