G.P.147 - Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: implications for clinical trials

Ricotti, V., Eagle, M., Butler, J., Decostre, V., Deborah, R., Moraux, A., Anthony, K., Sleby, V., Guglieri, M., Van der Holst, M., Jansen, M., Morgan, J., de Groot, I., Niks, E., Verschuuren, J., Servais, L., Hogrel, J. Y., Voit, T., Straub, V. and Muntoni, F. (2015) G.P.147 - Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: implications for clinical trials. Neuromuscular Disorders. 25(Supp 2) 0960-8966.

Ricotti_NM_2016_G.P.147_Outcome_measures_for_Duchenne_muscular_dystrophy_from_ambulant_to_non_ambulant_imp ... (80kB)

Item Type:	Article
Abstract:	Novel emerging therapies for Duchenne muscular dystrophy (DMD), such as antisense oligomer (AO) mediated exon skipping, have generated the need of understanding the natural history study of the targeted genotype subgroups. Most natural history studies are focused on ambulant subjects; therefore very little data exists on non-ambulant DMD. Specifically targeting skippable deletions, we aim to assess the natural history of DMD through a composite assessment tool capable of capturing disease progression beyond loss of ambulation.
Creators:	Ricotti, V, Eagle, M, Butler, J, Decostre, V, Deborah, R, Moraux, A, Anthony, Karen, Sleby, V, Guglieri, M, Van der Holst, M, Jansen, M, Morgan, J, de Groot, I, Niks, E, Verschuuren, J, Servais, L, Hogrel, J Y, Voit, T, Straub, V and Muntoni, F
Faculties, Divisions and Institutes:	Research Centres > Centre for Physical Activity and Life Sciences Faculties > Faculty of Health & Society > Sports, Exercise & Life Sciences Research Centres > Northamptonshire Dementia Research & Innovation Centre
Date:	1 October 2015
Date Type:	Publication
Journal or Publication Title:	Neuromuscular Disorders
Volume:	25
Number:	Supp 2
Language:	English
ISSN:	0960-8966
Status:	Published / Disseminated
Refereed:	Yes
Related URLs:	https://pure.northampton.ac.uk/en/public...
URI:	http://nectar.northampton.ac.uk/id/eprint/11658

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