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Translational Research in Europe for theAssessment and Treatment for NeuromuscularDisorders (TREAT-NMD)

Lookup NU author(s): Becca LearyORCiD, Dr Anne Oyewole, Emerita Professor Katherine Bushby, Professor Annemieke Aartsma-Rus

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Abstract

Translational research in Europe for the assessment and treatment of neuromusculardisorders (TREAT-NMD) is a global network of world-class experts within the neuromuscularcommunity whose mission is to support all stages of therapy developmentand improve the health and quality of life of people around the world with neuromusculardisorders (NMD). Since 2007, TREAT-NMD has played a central role in bringingtogether the right experts, patients, advocacy organizations, scientists, healthcareprofessionals, and pharmaceutical companies. By uniting these experts within theneuromuscular community, TREAT-NMD has supported preclinical research in animaland cell models that has raised the potential for clinical trials. TREAT-NMD hassuccessfully developed several key resources, including cell and animal standardoperating protocols (preclinical research), global patient registries, ethical frameworkand care guidelines, and family guides, to help develop and extend translation researchin the field, thereby making the field ready for clinical trials. This review aims tohighlight key achievements of the network, which spans a decade and the successfulpartnership with our stakeholders who have enabled the field to identify and addresschallenges to therapeutic development and standard of care for patients. This reviewwill also outline future perspectives.

Publication metadata

Author(s): Leary R, Oyewole A, Bushby K, Aarstma-Rus A

Publication type: Article

Publication status: Published

Journal: Neuropediatrics

Year: 2017

Volume: 48

Pages: 211-220

Print publication date: 29/05/2017

Online publication date: 06/07/2017

Acceptance date: 28/05/2017

ISSN (print): 0174-304X

ISSN (electronic): 1439-1899

Publisher: Georg Thieme Verlag KG

URL: https://doi.org/10.1055/s-0037-1604110

DOI: 10.1055/s-0037-1604110

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