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Abstracts & Posters

DMD clinical therapies II: P.135 DMD-HUB: expanding clinical trial capacity for Duchenne muscular dystrophy, 1 year on

key information

source: Neuromuscular Disorders

year: 2018

authors: E. Heslop, M. Guglieri, K. Bushby, C. Turner, B. Crow, E. Crossley, A. Johnson, M. Scoto, F. Muntoni, V. Straub

summary/abstract:

The need to increase capacity for Duchenne muscular dystrophy (DMD) trials and improve trial readiness was identified. Specifically, clinicians in established UK clinical trial centres involved in multiple DMD studies were reaching capacity, while centers with capacity lacked the expertise and needed support to develop and achieve the requirements set by industry to run clinical trials.

The DMD-Hub was set up as a unique partnership between Duchenne UK and two neuromuscular centres of excellence, The John Walton muscular dystrophy research centre in Newcastle and Great Ormond Street Institute of Child Health in London to address the issues together, share expertise and develop a network of trial ready centres able to take on interventional trials in DMD.

organization: Newcastle University, UK; Duchenne UK, UK; University College London, UK

DOI: 10.1016/j.nmd.2018.06.163

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