209th ENMC International Workshop: Outcome Measures and Clinical Trial Readiness in Spinal Muscular Atrophy 7-9 November 2014, Heemskerk, The Netherlands

被引：160
作者：
Finkel, Richard [1 ]
Bertini, Enrico [2 ]
Muntoni, Francesco [3 ]
Mercuri, Eugenio [4 ]
机构：
[1] Nemours Childrens Hosp, Orlando, FL 32827 USA
[2] UCL Inst Child Hlth, Dubowitz Neuromuscular Ctr, London, England
[3] Bambino Gesu Childrens Res Hosp, Rome, Italy
[4] Univ Cattolica Sacro Cuore, Pediat Neurol Unit, I-00168 Rome, Italy
来源：
NEUROMUSCULAR DISORDERS | 2015年 / 25卷 / 07期
关键词：
FUNCTIONAL MOTOR SCALE; PLACEBO-CONTROLLED TRIAL; SMN2 COPY NUMBER; DOUBLE-BLIND; MOUSE MODEL; YOUNG-CHILDREN; WALK TEST; SMA; RELIABILITY; VALIDATION;
D O I：
10.1016/j.nmd.2015.04.009
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
[No abstract available]
引用
收藏
页码：593 / 602
页数：10
相关论文
共 63 条
  • [1] Electrophysiological biomarkers in spinal muscular atrophy: proof of concept
    Arnold, W. David
    Porensky, Paul N.
    McGovern, Vicki L.
    Iyer, Chitra C.
    Duque, Sandra
    Li, Xiaobai
    Meyer, Kathrin
    Schmelzer, Leah
    Kaspar, Brian K.
    Kolb, Stephen J.
    Kissel, John T.
    Burghes, Arthur H. M.
    [J]. ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, 2014, 1 (01): : 34 - 44
  • [2] A motor function measure scale for neuromuscular diseases.: Construction and validation study
    Bérard, C
    Payan, C
    Hodgkinson, L
    Fermanian, J
    [J]. NEUROMUSCULAR DISORDERS, 2005, 15 (07) : 463 - 470
  • [3] Survival motor neuron protein deficiency impairs myotube formation by altering myogenic gene expression and focal adhesion dynamics
    Bricceno, Katherine V.
    Martinez, Tara
    Leikina, Evgenia
    Duguez, Stephanie
    Partridge, Terence A.
    Chernomordik, Leonid V.
    Fischbeck, Kenneth H.
    Sumner, Charlotte J.
    Burnett, Barrington G.
    [J]. HUMAN MOLECULAR GENETICS, 2014, 23 (18) : 4745 - 4757
  • [4] Abnormal motor phenotype in the SMNΔ7 mouse model of spinal muscular atrophy
    Butchbach, Matthew E. R.
    Edwards, Jonathan D.
    Burghes, Arthur H. M.
    [J]. NEUROBIOLOGY OF DISEASE, 2007, 27 (02) : 207 - 219
  • [5] RASCH ANALYSIS OF CLINICAL OUTCOME MEASURES IN SPINAL MUSCULAR ATROPHY
    Cano, Stefan J.
    Mayhew, Anna
    Glanzman, Allan M.
    Krosschell, Kristin J.
    Swoboda, Kathryn J.
    Main, Marion
    Steffensen, Birgit F.
    Berard, Carole
    Girardot, Francoise
    Payan, Christine A. M.
    Mercuri, Eugenio
    Mazzone, Elena
    Elsheikh, Bakri
    Florence, Julaine
    Hynan, Linda S.
    Iannaccone, Susan T.
    Nelson, Leslie L.
    Pandya, Shree
    Rose, Michael
    Scott, Charles
    Sadjadi, Reza
    Yore, Mackensie A.
    Joyce, Cynthia
    Kissel, John T.
    [J]. MUSCLE & NERVE, 2014, 49 (03) : 422 - 430
  • [6] Randomized, double-blind, placebo-controlled trial of hydroxyurea in spinal muscular atrophy
    Chen, T. -H.
    Chang, J. -G.
    Yang, Y. -H.
    Mai, H. -H.
    Liang, W. -C.
    Wu, Y. -C.
    Wang, H. -Y.
    Huang, Y. -B.
    Wu, S. -M.
    Chen, Y. -C.
    Yang, S. -N.
    Jong, Y. -J.
    [J]. NEUROLOGY, 2010, 75 (24) : 2190 - 2197
  • [7] Reliability and Validity of Outcome Measures of In-Hospital and At-Home Visits in a Randomized, Double-Blind, Placebo-Controlled Trial for Spinal Muscular Atrophy
    Chen, Tai-Heng
    Yang, Yi-Hsin
    Mai, Hui-Hsia
    Liang, Wen-Chen
    Wu, Yi-Ching
    Wang, Hui-Yi
    Jong, Yuh-Jyh
    [J]. JOURNAL OF CHILD NEUROLOGY, 2014, 29 (12) : 1680 - 1684
  • [8] Evaluation of SMN Protein, Transcript, and Copy Number in the Biomarkers for Spinal Muscular Atrophy (BforSMA) Clinical Study
    Crawford, Thomas O.
    Paushkin, Sergey V.
    Kobayashi, Dione T.
    Forrest, Suzanne J.
    Joyce, Cynthia L.
    Finkel, Richard S.
    Kaufmann, Petra
    Swoboda, Kathryn J.
    Tiziano, Danilo
    Lomastro, Rosa
    Li, Rebecca H.
    Trachtenberg, Felicia L.
    Plasterer, Thomas
    Chen, Karen S.
    [J]. PLOS ONE, 2012, 7 (04):
  • [9] Evaluation of muscle strength and motor abilities in children with type II and III spinal muscle atrophy treated with valproic acid
    Darbar, Illora A.
    Plaggert, Paulo G.
    Resende, Maria Bernadete D.
    Zanoteli, Edmar
    Reed, Umbertina C.
    [J]. BMC NEUROLOGY, 2011, 11
  • [10] Motor Function Measure: Validation of a Short Form for Young Children With Neuromuscular Diseases
    de Lattre, Capucine
    Payan, Christine
    Vuillerot, Carole
    Rippert, Pascal
    de Castro, Denis
    Berard, Carole
    Poirot, Isabelle
    [J]. ARCHIVES OF PHYSICAL MEDICINE AND REHABILITATION, 2013, 94 (11): : 2218 - 2226
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