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

V Ricotti, M Eagle, J Butler, V Decostre, R Deborah, A Moraux, Karen Anthony, V Sleby, M Guglieri, M Van der Holst, M Jansen, J Morgan, I de Groot, E Niks, J Verschuuren, L Servais, J Y Hogrel, T Voit, V Straub, F Muntoni

Research output: Contribution to JournalAbstract

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.
Original languageEnglish
JournalNeuromuscular Disorders
Volume25
Issue numberSupp 2
Publication statusPublished - 1 Oct 2015

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    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., & 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).