CRISPR

CRISPR for Duchenne Genome editing technology to correct the mutated dystrophin gene Duchenne muscular dystrophy is caused by a mutation in the dystrophin gene, which results in the absence of full-length dystrophin protein, needed to protect muscles. Without this...

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,...