Promoting meaningful clinical trial outcome measures for Duchenne muscular dystrophy

被引:3
|
作者
Peav, H. [1 ]
Kennedy, A. [2 ]
Fischer, R.
Bronson, A.
Furlong, P.
机构
[1] RTI Int, Res Triangle Pk, NC USA
[2] Parent Project Muscular Dystrophy, Hackensack, NJ USA
关键词
D O I
10.1016/j.nmd.2016.06.366
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
P.337
引用
收藏
页码:S187 / S187
页数:1
相关论文
共 50 条
  • [1] Outcome Measures in Duchenne Muscular Dystrophy
    Benemei, Silvia
    Gatto, Francesca
    Boni, Luca
    Pane, Marika
    [J]. ACTA NEUROLOGICA BELGICA, 2024,
  • [2] Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy
    Ricci, Giulia
    Bello, Luca
    Torri, Francesca
    Schirinzi, Erika
    Pegoraro, Elena
    Siciliano, Gabriele
    [J]. NEUROLOGICAL SCIENCES, 2022, 43 (Suppl 2) : 625 - 633
  • [3] Therapeutic opportunities and clinical outcome measures in Duchenne muscular dystrophy
    Giulia Ricci
    Luca Bello
    Francesca Torri
    Erika Schirinzi
    Elena Pegoraro
    Gabriele Siciliano
    [J]. Neurological Sciences, 2022, 43 : 625 - 633
  • [4] Determination of the minimal clinically important difference (MCID) for clinical trial outcome measures in Duchenne muscular dystrophy
    Pitchforth, J.
    Domingos, J.
    Iodice, M.
    Mayhew, A.
    Muntoni, F.
    [J]. NEUROMUSCULAR DISORDERS, 2018, 28 : S124 - S124
  • [5] Seated Outcome Measures in Children With Duchenne Muscular Dystrophy
    Kern, Rebecca
    Carvell, Kimberly
    Gupta, Apeksha
    Verma, Sumit
    [J]. PEDIATRIC PHYSICAL THERAPY, 2022, 34 (03) : 375 - 380
  • [6] SEATED OUTCOME MEASURES IN CHILDREN WITH DUCHENNE MUSCULAR DYSTROPHY
    Kern, Rebecca
    Verma, Sumit
    Carvell, Kimberly
    Gupta, Apeksha
    [J]. MUSCLE & NERVE, 2022, 66 : S102 - S102
  • [7] OUTCOME MEASURES FOR DUCHENNE MUSCULAR DYSTROPHY: IMPLICATIONS FOR TRIALS
    Domingos, Joana
    Eagle, Michelle
    Moraux, Amelie
    Butler, Jordan
    Decostre, Valerie
    Ridout, Deborah
    Mayhew, Anna
    Selby, Victoria
    Guglieri, Michela
    Van der Holst, Menno
    Jansen, Merel
    Verschuuren, Jan G. M.
    de Groot, Imelda
    Niks, Erik
    Servais, Laurent
    Hogrel, Jean-Yves
    Straub, Volker
    Voit, Thomas
    Ricotti, Valeria
    Muntoni, Francesco
    [J]. JOURNAL OF NEUROLOGY NEUROSURGERY AND PSYCHIATRY, 2017, 88 : A63 - A63
  • [8] Reliable surrogate outcome measures in multicenter clinical trials of duchenne muscular dystrophy
    Mayhew, Jill E.
    Florence, Julaine M.
    Mayhew, Thomas P.
    Henricson, Erik K.
    Leshner, Robert T.
    McCarter, Robert J.
    Escolar, Diana M.
    [J]. MUSCLE & NERVE, 2007, 35 (01) : 36 - 42
  • [9] Determination of the minimal clinically important difference (MCID) for clinical trial outcome measures in Duchenne Muscular Dystrophy (DMD)
    Pitchforth, J. M.
    Domingos, J.
    Iodice, M.
    Mayhew, A.
    Muntoni, F.
    [J]. NEUROMUSCULAR DISORDERS, 2018, 28 : S17 - S17
  • [10] Outcome measures in Duchenne muscular dystrophy: sensitivity to change, clinical meaningfulness, and implications for clinical trials
    Domingos, Joana
    Muntoni, Francesco
    [J]. DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY, 2018, 60 (02): : 117 - 117