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), S229. 0960-8966.

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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.

Subjects:

R Medicine > RC Internal medicine > RC925 Diseases of the musculoskeletal system > RC935.M7 Muscular dystrophy
Q Science > QH Natural history > QH426 Genetics > QH438.7 Human genetics

Creators:

Publisher:

Elsevier

Faculties, Divisions and Institutes:

University Faculties, Divisions and Research Centres - OLD > Research Centre > Institute of Health and Wellbeing > Ageing Research Centre
Research Centres > Centre for Health Sciences and Services
Research Centres > Centre for Physical Activity and Life Sciences

Date:

October 2015

Date Type:

Publication

Page Range:

S229

Journal or Publication Title:

Neuromuscular Disorders

Volume:

Number:

Supp 2

Language:

English

DOI:

10.1016/j.nmd.2015.06.162

ISSN:

0960-8966

Status:

Published / Disseminated