The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials

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The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials

Ricotti, V; Ridout, DA; Pane, M; Main, M; Mayhew, A; Mercuri, E; Manzur, AY; ... on behalf of UK NorthStar Clinical Network, .; + view all (2016) The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials. Journal of Neurology, Neurosurgery & Psychiatry , 87 pp. 149-155. 10.1136/jnnp-2014-309405. Green open access

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Abstract

With the emergence of experimental therapies for Duchenne muscular dystrophy (DMD), it is fundamental to understand the natural history of this disorder to properly design clinical trials. The aims of this study were to assess the effects produced on motor function by different DMD genotypes and early initiation of glucocorticoids.

Type:	Article
Title:	The NorthStar Ambulatory Assessment in Duchenne muscular dystrophy: considerations for the design of clinical trials
Open access status:	An open access version is available from UCL Discovery
DOI:	10.1136/jnnp-2014-309405
Publisher version:	http://dx.doi.org/10.1136/jnnp-2014-309405
Language:	English
Additional information:	This is an Open Access article distributed in accordance with the Creative Commons Attribution Non Commercial (CC BY-NC 4.0) license, which permits others to distribute, remix, adapt, build upon this work non-commercially, and license their derivative works on different terms, provided the original work is properly cited and the use is non-commercial. See: http://creativecommons.org/licenses/by-nc/4.0/
Keywords:	MUSCULAR DYSTROPHY, NEUROMUSCULAR
UCL classification:	UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Brain Sciences > UCL Queen Square Institute of Neurology > Department of Neuromuscular Diseases UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Developmental Neurosciences Dept UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > UCL GOS Institute of Child Health > Population, Policy and Practice Dept
URI:	https://discovery.ucl.ac.uk/id/eprint/1464591

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