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 journalAbstractResearchpeer-review

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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Duchenne Muscular Dystrophy
Outcome Assessment (Health Care)
Clinical Trials
Natural History
Walking
Disease Progression
Exons
Genotype
Therapeutics

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Ricotti, V., Eagle, M., Butler, J., Decostre, V., Deborah, R., Moraux, A., ... 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).
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 ; Muntoni, F. / G.P.147 - Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: implications for clinical trials. In: Neuromuscular Disorders. 2015 ; Vol. 25, No. Supp 2.
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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.",
author = "V Ricotti and M Eagle and J Butler and V Decostre and R Deborah and A Moraux and Karen Anthony and V Sleby and M Guglieri and {Van der Holst}, M and M Jansen and J Morgan and {de Groot}, I and E Niks and J Verschuuren and L Servais and Hogrel, {J Y} and T Voit and V Straub and F Muntoni",
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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, JY, 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, vol. 25, no. Supp 2.

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, 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; Muntoni, F.

In: Neuromuscular Disorders, Vol. 25, No. Supp 2, 01.10.2015.

Research output: Contribution to journalAbstractResearchpeer-review

TY - JOUR

T1 - G.P.147 - Outcome measures for Duchenne muscular dystrophy from ambulant to non-ambulant: implications for clinical trials

AU - Ricotti, V

AU - Eagle, M

AU - Butler, J

AU - Decostre, V

AU - Deborah, R

AU - Moraux, A

AU - Anthony, Karen

AU - Sleby, V

AU - Guglieri, M

AU - Van der Holst, M

AU - Jansen, M

AU - Morgan, J

AU - de Groot, I

AU - Niks, E

AU - Verschuuren, J

AU - Servais, L

AU - Hogrel, J Y

AU - Voit, T

AU - Straub, V

AU - Muntoni, F

PY - 2015/10/1

Y1 - 2015/10/1

N2 - 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.

AB - 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.

M3 - Abstract

VL - 25

JO - Neuromuscular Disorders

JF - Neuromuscular Disorders

SN - 0960-8966

IS - Supp 2

ER -