GOSH coordinator

Great Ormond Street Hospital Clinical Trial Co-ordinator Manju Agarwal As part of the Newcastle Plan in 2015/16, eight patient organisations contributed £1.2m to fund at least 16 vital posts across the UK to support and expand clinical trial capacity for Duchenne...

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...

Newcastle CRF refurbishment

Refurbishment and expansion of paediatric space Clinical Research Facility at Newcastle Royal Infirmary The Duchenne Research Fund, Duchenne UK and Joining Jack funded the recent refurbishment and expansion of the paediatric area in the Clinical Research Facility at...

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...