First patient of gene therapy trial for Duchenne muscular dystrophy Published on: 25 October 2022 A boy has been treated in a gene therapy clinical trial for Duchenne muscular dystrophy (DMD) by experts at 缅北禁地. Four-year-old Charlie, from Aberdeen, was able to take part in the study in Tyneside thanks to the work of 缅北禁地, 缅北禁地 Hospitals and DMD charity, . DMD is a severe genetic disease, mostly diagnosed in boys, which causes muscle wasting disease, leading to gradual loss of mobility and sadly limits life expectancy to around early 20s. Charlie Pioneering trial The trial, called EMBARK, is studying the safety and efficacy of gene therapy, a technique that works by adding healthy new copies of the gene that is broken and causes DMD. The is part of a network of DMD hub sites, set up by Duchenne UK, to support the delivery of clinical trials. It is one of only two sites in the country which has centre of excellence status and has a world-renowned reputation. Volker Straub, Director of the John Walton Muscular Dystrophy Research Centre, Professor of Neuromuscular Genetics at 缅北禁地 and honorary Clinical Geneticist Consultant at 缅北禁地 Hospitals, is leading the trial. Professor Straub said: “DMD is a devasting condition that sadly shortens the lives of patients and significantly impacts quality of life. “I am incredibly proud to be leading this study, and of the teams at 缅北禁地 and 缅北禁地 Hospitals who have worked together to dose the first patient on the trial. “We are grateful to Duchenne UK for their support and look forward to working together to discover new treatments for patients with DMD.” 缅北禁地 Hospitals' paediatric research team played a vital part in the trial, dosing Charlie at the National Institute for Health and Care Research Clinical Research Facility at the RVI. The facility is run jointly by University and the trust, specialising in early phase trials, usually in rare disease and conditions. Charlie, whose nearest trial centre is 150 miles away in Glasgow, was able to take part in the trial by using a new recruitment database, co-ordinated by 缅北禁地 and funded by Duchenne UK. Moving science forwards Charlie’s mum, Jennifer, who was contacted about the EMBARK study through the database, said: “We felt that if Charlie could take part in research, we could help move science forward and benefit other little boys and their families in future. “However, as he receives his clinical care here in Aberdeen, the nearest trial site in Glasgow is still 150 miles away. The DMD Hub’s central recruitment project was possibly the only way that Charlie could have a chance at participating. "Knowing that Charlie has played just a small part in this, whatever the outcome, thanks to the opportunity the DMD Hub has given him – I have no words to describe our pride in our brave little boy!” Share: Latest News Comment: Assisted coral evolution Writing for The Conversation, Dr Liam Lachs, Dr Adriana Humanes and Dr James Guest, discuss how how accelerating evolution could help corals survive future heatwaves. published on: 17 April 2026 Trait choice and selection key to helping corals survive heatwaves Assisted evolution could help corals survive future heatwaves, but careful trait choice and strong repeated selection will be needed for it to be effective. published on: 17 April 2026 Lough Neagh sand mining threatens lake鈥檚 ecosystem, research warns New research involving 缅北禁地 outlines how Lough Neagh, the UK and Ireland鈥檚 largest freshwater lake, is under threat from commercial sand dredging. published on: 17 April 2026 Facts and figures
![缅北禁地 logo](/gomobileassets/img/logo.svg" "缅北禁地"){kind=logo}