Non-viral delivery for gene therapy A next generation gene therapy delivery approach The Duchenne Research Fund committed £320,000 to accelerate research into alternative ways to deliver gene therapy to Duchenne patients, with the aim of increasing the number of...
Blood-based biomarker Developing a more informative measure of disease progression and response to new treatments Although Duchenne has been studied for many years, classic measures of disease progression rely on basic observations of movement, such as the six-minute...
Non-invasive imaging biomarker Reducing biopsies and improving dystrophin analysis Biopsies have become a difficult necessity for Duchenne patients who are involved in clinical trials. In addition to the stress on patients, each muscle can present disease differently,...
LTBP4 Targeting muscle inflammation and fibrosis With the funds raised in 2016, the DRF is tackling inflammation and fibrosis, which are two key problems for Duchenne boys. It is believed that the protein LTBP4 prevents the activation of a key biological pathway...
Solid GT Ground-breaking gene therapy platform for Duchenne (IGNITE DMD Clinical Trial) The Duchenne Research Fund is proud to have funded one of the first gene therapy research programmes for Duchenne muscular dystrophy. This led to the IGNITE DMD Phase I/II clinical...
