Nusinersen Efficacy in Adults with Spinal Muscular Atrophy

被引:0
|
作者
Day, John [1 ]
Wolford, Connie [2 ]
MacPherson, Chelsea [4 ]
Martens, William [5 ]
McDermott, Michael [6 ]
Darras, Basil [7 ]
De Vivo, Daryl [8 ]
Cunningham, Zarazuela Zolkipli [9 ]
Finkel, Richard [10 ]
Sampson, Jacinda [3 ]
Duong, Tina [1 ]
机构
[1] Stanford Univ, Sch Med, Palo Alto, CA 94304 USA
[2] Stanford Univ, Neurol, Stanford, CA 94305 USA
[3] Stanford Univ, Fremont, CA USA
[4] Stanford Healthcare, Rehabil, Stanford, CA USA
[5] Univ Rochester, Rochester, NY USA
[6] Rochester Univ, Rochester, NY USA
[7] Harvard Med Sch, Childrens Hosp Boston, Boston, MA USA
[8] Columbia Univ, Neurol, Boston, MA USA
[9] Childrens Hosp Philadelphia, Neurol, Philadelphia, PA 19104 USA
[10] Nemours Childrens Hosp, Orlando, FL USA
关键词
D O I
暂无
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
S46.001
引用
收藏
页数:3
相关论文
共 50 条
  • [41] Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
    David R Corey
    [J]. Nature Neuroscience, 2017, 20 : 497 - 499
  • [42] Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
    Corey, David R.
    [J]. NATURE NEUROSCIENCE, 2017, 20 (04) : 497 - 499
  • [43] Prospective open-label study of Nusinersen treatment for adults with Spinal Muscular Atrophy
    Elsheikh, Bakri
    Severyn, Steven
    Arnold, William
    Kolb, Stephen
    Kissel, John
    [J]. NEUROLOGY, 2019, 92 (15)
  • [44] Sometimes they come back: New and old spinal muscular atrophy adults in the era of nusinersen
    Sansone, V. A.
    Coratti, G.
    Pera, M. C.
    Pane, M.
    Messina, S.
    Salmin, F.
    Albamonte, E.
    De Sanctis, R.
    Sframeli, M.
    Di Bella, V
    Morando, S.
    D'Amico, A.
    Frongia, A. L.
    Antonaci, L.
    Pirola, A.
    Pedemonte, M.
    Bertini, E.
    Bruno, C.
    Mercuri, E.
    [J]. EUROPEAN JOURNAL OF NEUROLOGY, 2021, 28 (02) : 602 - 608
  • [45] Nusinersen injections in adults and children with spinal muscular atrophy: a single-center experience
    Ozutemiz, Can
    Karachunski, Peter
    Nascene, David R.
    [J]. DIAGNOSTIC AND INTERVENTIONAL RADIOLOGY, 2020, 26 (06) : 596 - 602
  • [46] Safety, Tolerability, and Effect of Nusinersen in Non-ambulatory Adults With Spinal Muscular Atrophy
    Elsheikh, Bakri
    Severyn, Steven
    Zhao, Songzhu
    Kline, David
    Linsenmayer, Matthew
    Kelly, Kristina
    Tellez, Marco
    Bartlett, Amy
    Heintzman, Sarah
    Reynolds, Jerry
    Sterling, Gary
    Weaver, Tristan
    Rajneesh, Kiran
    Kolb, Stephen J.
    Arnold, W. David
    [J]. FRONTIERS IN NEUROLOGY, 2021, 12
  • [47] Nusinersen: A Review in 5q Spinal Muscular Atrophy
    Sheridan M. Hoy
    [J]. CNS Drugs, 2021, 35 : 1317 - 1328
  • [48] Study on the efficacy, safety, and biomarkers of nusinersen in type II and III spinal muscular atrophy in children
    Chen, Liyuan
    Liu, Fen
    Fang, Danna
    Li, Jianwei
    [J]. FRONTIERS IN PEDIATRICS, 2023, 11
  • [49] Respiratory assessment in a spinal muscular atrophy infant treated with nusinersen
    Ogawa, Kazuya
    Okanari, Kazuo
    Kobayashi, Osamu
    Nakashima, Misaki
    Ihara, Kenji
    [J]. PEDIATRICS INTERNATIONAL, 2019, 61 (10) : 1051 - 1053
  • [50] Evaluation of Metabolic Effects of Nusinersen in Patients with Spinal Muscular Atrophy
    Becker, Lena-Luise
    Weiss, Claudia
    Guenther, Rene
    Hermann, Andreas
    Theophil, Manuela
    Huebner, Angela
    Smitka, Martin
    von der Hagen, Maja
    Kaindl, Angela M.
    [J]. JOURNAL OF PEDIATRIC NEUROLOGY, 2022, 20 (04) : 252 - 257