Access to treatments

Duchenne UK's DMD HUB unveiled at World Muscle Society Meeting

Emma Heslop, the DMD HUB manager, is pictured presenting the DMD Hub poster at 22nd International Congress of The World Muscle Society which took place in October.

The DMD Hub is a network of trial sites - and staff - trained and funded to carry out clinical trials for Duchenne Muscular Dystrophy (DMD). The Hub has been conceived and developed in collaboration with leading neuromuscular clinicians.

The Hub’s aim, is to make more clinical sites trial ready by providing training, mentoring and resources to help them achieve the requirements set by industry to run clinical trials.

Duchenne UK’s mission for the DMD Hub is to ensure that all patients with DMD, including children and
adults, have the opportunity to access clinical research opportunities.

The below abstract on the DMD Hub was included in: Neuromuscular Disorders Volume 27, Supplement 2, which can be read below or on the website here.

DMD HUB: Expanding clinical trial capacity for Duchenne muscular dystrophy in the UK

E. Heslop 1, V. Straub 1, M. Guglieri 1, C. Turner 1, B. Davis 1, E. Crossley 2, A. Johnson 2, K. Bushby 1

1 John Walton Muscular Dystrophy Research Centre, Newcastle University, Newcastle upon Tyne, UK; 2 Duchenne UK, London, UK

With Duchenne muscular dystrophy (DMD) clinical research at an unprecedented stage in terms of the number of possible therapeutic approaches coming to trials, a workshop was organized to bring together 75 participants from the DMD patient and research community, as well as industry to address concerns regarding the apparent lack of capacity for trials in the UK. Specifically, clinicians in established UK clinical trial centres involved in multiple DMD studies are reaching capacity, while centres that have capacity but lack the expertise need support to develop to achieve the requirements set by industry to run clinical trials. A key outcome of the workshop was the recommendation to establish a UK Hub to expand the clinical trial capacity for DMD. The Hub started in 2016, funded by Duchenne UK, and we are now working in conjunction with staff and facilities available at sites to implement a network of UK sites, providing expert DMD advice, guidance, and training to enable them to take on trials thereby relieving pressure on experienced sites. The Hub is collaborating with existing organisations such as TREAT-NMD, the National Institutes of Health Research, the North Star Network, patient advocacy groups and the DMD patient registry run by action Duchenne to avoid duplication of the significant work already achieved in this area. Within the first year of operation the Hub expects 2–3 new trial sites to be selected for industry led DMD trials in the UK while ongoing training for other sites should open opportunities even further in subsequent years. The mission of the DMD Hub ultimately is to ensure all patients with DMD, including children and adults, have access to clinical research opportunities. Issues of sustainability and expansion of the model to other rare neuromuscular diseases are being explored.

Published on 11 January 2017

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