Gene therapy for Becker

Gene therapy for Becker A microdystrophin gene therapy approach for Becker muscular dystrophy We have awarded a grant to the team of Professor Thomas Voit (pictured) at the Great Ormond Street Biomedical Research Centre at UCL to develop a microdystrophin gene therapy...

Activity monitoring biomarker – KineDMD

KINEDMD study: developing an activity monitoring biomarker Using novel algorithms to chart daily activities of ambulant and non-ambulant boys The KineDMD study overseen by Imperial College London is now open for recruitment. The study will recruit 16 Duchenne muscular...

Blood-based biomarker

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

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

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...