Arbaclofen in fragile X syndrome: results of phase 3 trials

被引:0
|
作者
Elizabeth Berry-Kravis
Randi Hagerman
Jeannie Visootsak
Dejan Budimirovic
Walter E. Kaufmann
Maryann Cherubini
Peter Zarevics
Karen Walton-Bowen
Paul Wang
Mark F. Bear
Randall L. Carpenter
机构
[1] Rush University Medical Center,Departments of Pediatrics, Neurological Sciences, Biochemistry
[2] University of California Davis Medical Center,MIND Institute and Department of Pediatrics
[3] Emory University,Department of Human Genetics
[4] Kennedy Krieger Institute,Departments of Psychiatry &Behavioral Sciences
[5] the Johns Hopkins Medical Institutions,Department of Neurology, Boston Children’s Hospital
[6] Boston,undefined
[7] MA 02115 and Greenwood Genetic Center,undefined
[8] Seaside Therapeutics Inc,undefined
[9] Simons Foundation Autism Research Initiative,undefined
[10] Autism Speaks,undefined
[11] The Picower Institute for Learning and Memory,undefined
[12] Massachusetts Institute of Technology,undefined
[13] Rett Syndrome Research Trust,undefined
来源
Journal of Neurodevelopmental Disorders | 2017年 / 9卷
关键词
Fragile X syndrome; Arbaclofen; GABA agonist; FMR1; Targeted treatment; Neurodevelopmental disorder;
D O I
暂无
中图分类号
学科分类号
摘要
引用
收藏
相关论文
共 50 条
  • [1] Arbaclofen in fragile X syndrome: results of phase 3 trials
    Berry-Kravis, Elizabeth
    Hagerman, Randi
    Visootsak, Jeannie
    Budimirovic, Dejan
    Kaufmann, Walter E.
    Cherubini, Maryann
    Zarevics, Peter
    Walton-Bowen, Karen
    Wang, Paul
    Bear, Mark F.
    Carpenter, Randall L.
    JOURNAL OF NEURODEVELOPMENTAL DISORDERS, 2017, 9
  • [2] Targeted treatment for fragile X syndrome: results of a randomized controlled phase II trial of arbaclofen
    Hagerman, Randi
    Rathmell, Barbara
    Wang, Lulu
    Carpenter, Randy
    Wang, Paul
    Berry-Kravis, Elizabeth
    JOURNAL OF INTELLECTUAL DISABILITY RESEARCH, 2010, 54 : 884 - 884
  • [3] The challenges of clinical trials in fragile X syndrome
    Jacquemont, Sebastien
    Berry-Kravis, Elizabeth
    Hagerman, Randi
    von Raison, Florian
    Gasparini, Fabrizio
    Apostol, George
    Ufer, Mike
    Des Portes, Vincent
    Gomez-Mancilla, Baltazar
    PSYCHOPHARMACOLOGY, 2014, 231 (06) : 1237 - 1250
  • [4] The challenges of clinical trials in fragile X syndrome
    Sébastien Jacquemont
    Elizabeth Berry-Kravis
    Randi Hagerman
    Florian von Raison
    Fabrizio Gasparini
    George Apostol
    Mike Ufer
    Vincent Des Portes
    Baltazar Gomez-Mancilla
    Psychopharmacology, 2014, 231 : 1237 - 1250
  • [5] Parent Ratings of Ability to Consent for Clinical Trials in Fragile X Syndrome
    Bailey, Donald B., Jr.
    Raspa, Melissa
    Wheeler, Anne
    Edwards, Anne
    Bishop, Ellen
    Bann, Carla
    Borasky, David
    Appelbaum, Paul S.
    JOURNAL OF EMPIRICAL RESEARCH ON HUMAN RESEARCH ETHICS, 2014, 9 (03) : 18 - 28
  • [6] Fragile X Syndrome
    Saldarriaga, Wilmar
    Tassone, Flora
    Yuriko Gonzalez-Teshima, Laura
    Vicente Forero-Forero, Jose
    Ayala-Zapata, Sebastian
    Hagerman, Randi
    COLOMBIA MEDICA, 2014, 45 (04): : 190 - 198
  • [7] The fragile X syndrome
    de Vries, BBA
    Halley, DJJ
    Oostra, BA
    Niermeijer, MF
    JOURNAL OF MEDICAL GENETICS, 1998, 35 (07) : 579 - 589
  • [8] The fragile X syndrome
    Szot, M
    FOLIA HISTOCHEMICA ET CYTOBIOLOGICA, 1997, 35 (03): : 135 - 141
  • [9] Fragile X syndrome
    Pimentel, MMG
    INTERNATIONAL JOURNAL OF MOLECULAR MEDICINE, 1999, 3 (06) : 639 - 645
  • [10] Fragile X syndrome
    Kathryn B Garber
    Jeannie Visootsak
    Stephen T Warren
    European Journal of Human Genetics, 2008, 16 : 666 - 672