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

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 Journal › Abstract › peer-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 language	English
Journal	Neuromuscular Disorders
Volume	25
Issue number	Supp 2
Publication status	Published - 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).

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

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",

year = "2015",

month = oct,

day = "1",

language = "English",

volume = "25",

journal = "Neuromuscular Disorders",

issn = "0960-8966",

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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.

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

SN - 0960-8966

VL - 25

JO - Neuromuscular Disorders

JF - Neuromuscular Disorders

IS - Supp 2

ER -

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

Abstract

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