Professor Dame Kay Davies at Oxford University is aiming to identify biomarkers that can monitor the effectiveness of utrophin up-regulators. These will provide a more objective measure for future clinical trials and will also help to accelerate the development of utrophin up-regulators.

Gene therapy offers the hope of delivering a fully functional dystrophin gene to muscles for people with Duchenne muscular dystrophy. In this project Professor George Dickson and his team will develop improved gene therapy techniques that have an enhanced ability to increase dystrophin protein in muscles. The researchers will do this by making changes to the micro-dystrophin gene and to the harmless virus that delivers it to muscles. This is a continuation of Professor Dickson’s previous work that could result in improved, second-generation gene therapies to test in future clinical trials, and better treatment options for people with Duchenne muscular dystrophy in the long term.

The Muscular Dystrophy Campaign funds two clinical trials coordinators who are crucial for the efficient running of clinical trials at the muscle centres in London and Newcastle.