Our projects
Brilliant scientists, powerful collaborations. We find and fund projects that are ripe for treating Duchenne now.CRISPR for Duchenne
Gene editing technology to correct mutated dystrophin gene.
Combating immunity to AAV
Ensuring as many patients as possible can benefit from gene therapy.
Solid GT
A ground-breaking programme for exploring gene therapy for Duchenne.
Vamorolone
An alternative to existing steroid treatments for Duchenne.
LTBP4
Targeting inflammation and fibrosis – two key challenges in Duchenne.
Gene therapy for Becker
A microdystrophin gene therapy approach for Becker muscular dystrophy.
Activity monitoring biomarker
KINEDMD: Wearable sensor technology to monitor Duchenne patients’ activity.
Non-viral delivery
A next-generation gene therapy delivery approach.
Non-invasive imaging biomarker
Reducing biopsies and improving dystrophin analysis in Duchenne boys.
Blood-based biomarker
Measuring disease progression and response to new treatments.
Grants to support clinical and research staff and facilities
Awards for up-and-coming researchers
Some of our past projects
Our commitment
We are passionate about supporting the most promising research to find therapies and a cure for Duchenne muscular dystrophy. We aim to focus our efforts on tackling Duchenne for all sufferers, regardless of their type of mutation. We have an open grant call and all applications are peer reviewed.
Our scientific advisory board comprises leading minds in the field: Professor Ronald Cohn, Dr Kevin Campbell, Professor Madeleine Durbeej-Hjalt and Dr James Dowling together with our Honorary Scientific Patron Professor Victor Dubowitz. Read more about their work here. Please contact research [at] duchenne.org.uk for more information about applications.
Our mission
We aim to stop Duchenne muscular dystrophy being a death sentence for hundreds of thousands of boys worldwide.
Our impact
We are helping turn the hope of treatments into reality. Critical supportive drugs are in clinical trial thanks to our funds.
Your help
In the last 4 years we have given more than £3m to ground-breaking research projects.
And there is still work to do.