Early Clinical Experience with Onasemnogene Abeparvovec in Spinal Muscular Atrophy (SMA) Type 2 Patients at a Single Center

被引：0
|
作者：
Matesanz, Susan
Kichula, Elizabeth
机构：
来源：
NEUROLOGY | 2020年 / 94卷 / 15期
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：
4855
引用
收藏
页数：3
相关论文
共 50 条
  • [1] Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy
    Hannah A. Blair
    CNS Drugs, 2022, 36 : 995 - 1005
  • [2] Onasemnogene Abeparvovec: A Review in Spinal Muscular Atrophy
    Blair, Hannah A.
    CNS DRUGS, 2022, 36 (09) : 995 - 1005
  • [3] Onasemnogene abeparvovec for the treatment of spinal muscular atrophy
    McMillan, Hugh J.
    Proud, Crystal M.
    Farrar, Michelle A.
    Alexander, Ian E.
    Muntoni, Francesco
    Servais, Laurent
    EXPERT OPINION ON BIOLOGICAL THERAPY, 2022, 22 (09) : 1075 - 1090
  • [4] Onasemnogene abeparvovec in spinal muscular atrophy: an Australian experience of safety and efficacy
    D'Silva, Arlene M.
    Holland, Sandra
    Kariyawasam, Didu
    Herbert, Karen
    Barclay, Peter
    Cairns, Anita
    MacLennan, Suzanna C.
    Ryan, Monique M.
    Sampaio, Hugo
    Smith, Nicholas
    Woodcock, Ian R.
    Yiu, Eppie M.
    Alexander, Ian E.
    Farrar, Michelle A.
    ANNALS OF CLINICAL AND TRANSLATIONAL NEUROLOGY, 2022, 9 (03): : 339 - 350
  • [5] Intravenous (IV) onasemnogene abeparvovec for spinal muscular atrophy (SMA): integrated safety report
    Chand, D.
    Finkel, R.
    Mercuri, E.
    Masson, R.
    Parsons, J.
    Kleyn, A.
    Menier, M.
    Montgomery, K.
    Sproule, D.
    Reyna, S.
    Feltner, D.
    Tauscher-Wisniewki, S.
    Mendell, J.
    NEUROMUSCULAR DISORDERS, 2020, 30 : S120 - S121
  • [6] Bulbar function for patients with spinal muscular atrophy type 1 following onasemnogene abeparvovec
    McGrattan, K.
    Shell, R.
    Hurst-Davis, R.
    Young, S. Dunaway
    O'Brien, E.
    Lavrov, A.
    Wallach, S.
    LaMarca, N.
    Reyna, S.
    Darras, B.
    NEUROMUSCULAR DISORDERS, 2022, 32 : S85 - S85
  • [7] Routine practices in use of onasemnogene abeparvovec (OA) in older patients with spinal muscular atrophy (SMA): Early findings from RESTORE
    Servais, L.
    De Vivo, D.
    Kirschner, J.
    Mercuri, E.
    Muntoni, F.
    Tizzano, E.
    Roy, S.
    Saito, K.
    Menier, M.
    LaMarca, N.
    Anderson, F.
    Dabbous, O.
    Finkel, R.
    NEUROMUSCULAR DISORDERS, 2021, 31 : S138 - S138
  • [8] Safety Monitoring of Gene Therapy for Spinal Muscular Atrophy with Onasemnogene Abeparvovec - A Single Centre Experience
    Friese, Johannes
    Geitmann, Stephanie
    Holzwarth, Dorothea
    Mueller, Nicole
    Sassen, Robert
    Baur, Ute
    Adler, Kristin
    Kirschner, Janbernd
    JOURNAL OF NEUROMUSCULAR DISEASES, 2021, 8 (02) : 209 - 216
  • [9] Routine Practices in use of Onasemnogene Abeparvovec (OA) in Older Patients with Spinal Muscular Atrophy (SMA): Early Findings from RESTORE
    不详
    ANNALS OF NEUROLOGY, 2021, 90 : S158 - S158
  • [10] Integrated Safety Report for Intravenous (IV) Onasemnogene Abeparvovec Clinical Development Programs in Spinal Muscular Atrophy (SMA) Type 1 (SMA1)
    Tauscher-Wisniewski, S.
    Finkel, R. S.
    Mercuri, E.
    Masson, R.
    Parsons, J.
    Baldinetti, F.
    Chand, D.
    Kleyn, A.
    Sproule, D. M.
    Feltner, D. E.
    Mendell, J. R.
    EUROPEAN JOURNAL OF NEUROLOGY, 2020, 27 : 875 - 875
12345