30 Nov 2017
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TREAT-NMD: Advancing Diagnosis, Treatment and Care in Neuromuscular Rare Diseases
Authors:
Rebecca Leary (becca.leary@ncl.ac.uk), Rebecca Leary 1,Anne Oyewole 1, Annemieke Aartsma-Rus1 2, and Katharine Bushby 1
on behalf of the TREAT-NMD Alliance
1 John Walton Muscular Dystrophy Research Centre, Institute of Genetic Medicine, Newcastle University, UK. 2 Leiden University Medical Center, Leiden, The Netherlands
Translational Research in Europe: Assessment and Treatment of Neuromuscular Disorders (TREAT-NMD) is a global network of world-class experts within the neuromuscular community that includes patients, advocacy organisations, scientists, clinicians and industry. Our mission is to speed up the process of developing new treatments and improve the health and quality of life for people around the world with NMD, including Duchenne muscular dystrophy (DMD) and Spinal muscular atrophy (SMA), by supporting all stages of translational research. Over the last decade we have identified major barriers in the diagnosis, treatment and care for patients with NMD and have developed essential ‘go-to’ tools and resources to overcome these challenges.
TREAT-NMD will be a key partner for EURO-NMD. The tools and resources developed over the last 10 years can be utilised by the ERN.