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
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
  • [41] Expert recommendations and clinical considerations in the use of onasemnogene abeparvovec gene therapy for spinal muscular atrophy
    Kichula, Elizabeth A.
    Proud, Crystal M.
    Farrar, Michelle A.
    Kwon, Jennifer M.
    Saito, Kayoko
    Desguerre, Isabelle
    McMillan, Hugh J.
    MUSCLE & NERVE, 2021, 64 (04) : 413 - 427
  • [42] Onasemnogene Abeparvovec for Presymptomatic Infants with Spinal Muscular Atrophy and Two Copies of SMN2
    Strauss, Kevin
    Muntoni, Francesco
    Farrar, Michelle
    Saito, Kayoko
    Mendell, Jerry
    Servals, Laurent
    McMillan, Hugh
    Finkel, Richard
    Swoboda, Kathryn
    Kwon, Jennifer
    Zaidman, Craig
    Chiriboga, Claudia
    Iannaccone, Susan
    Krueger, Jena
    Parsons, Julie
    Shieh, Perry
    Kavanagh, Sarah
    Chand, Deepa
    Tauscher-Wisniewski, Sitra
    McGill, Bryan
    Macek, Thomas
    NEUROLOGY, 2022, 98 (18)
  • [43] Long-term follow-up (LTFU) of onasemnogene abeparvovec gene therapy in spinal muscular atrophy (SMA)
    Mendell, J.
    Finkel, R.
    Mercuri, E.
    Strauss, K.
    Day, J.
    Kleyn, A.
    Chand, D.
    Tauscher-Wisniewski, S.
    Meriggioli, M.
    NEUROMUSCULAR DISORDERS, 2021, 31 : S132 - S133
  • [44] Effectiveness and Safety of Onasemnogene Abeparvovec in Older Patients with Spinal Muscular Atrophy (SMA): Real-World Outcomes from the RESTORE Registry
    Servais, Laurent
    De Vivo, Darryl
    Kirschner, Janbernd
    Mercuri, Eugenio
    Muntoni, Francesco
    Tizzano, Eduardo
    Quijano-Roy, Susana
    Saito, Kayoko
    Menier, Melissa
    LaMarca, Nicole
    Anderson, Fred
    Dabbous, Omar
    Finkel, Richard
    NEUROLOGY, 2022, 98 (18)
  • [45] Clinical features of spinal muscular atrophy (SMA) type 2
    Cances, C.
    Richelme, C.
    Barnerias, C.
    Espil, C.
    ARCHIVES DE PEDIATRIE, 2020, 27 (07): : 18 - 22
  • [46] Necrotizing Enterocolitis following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
    Gaillard, Jonathan
    Gu, Andrew Ran
    Knierbein, Erin E. Neil
    JOURNAL OF PEDIATRICS, 2023, 260
  • [47] Thrombotic Microangiopathy Following Onasemnogene Abeparvovec for Spinal Muscular Atrophy: A Case Series
    Chand, Deepa H.
    Zaidman, Craig
    Arya, Kapil
    Millner, Rachel
    Farrar, Michelle A.
    Mackie, Fiona E.
    Goedeker, Natalie L.
    Dharnidharka, Vikas R.
    Dandamudi, Raja
    Reyna, Sandra P.
    JOURNAL OF PEDIATRICS, 2021, 231 : 265 - 268
  • [48] Onasemnogene abeparvovec gene therapy in presymptomatic spinal muscular atrophy (SMA): SPR1NT study update
    Strauss, K.
    Farrar, M.
    Swoboda, K.
    Saito, K.
    Chiriboga, C.
    Finkel, R.
    Iannaccone, S.
    Krueger, J.
    Kwon, J.
    McMillan, H.
    Servais, L.
    Mendell, J.
    Parsons, J.
    Scoto, M.
    Shieh, P.
    Zaidman, C.
    Schultz, M.
    Ogrinc, F.
    Group, S.
    Muntoni, F.
    NEUROMUSCULAR DISORDERS, 2020, 30 : S122 - S122
  • [49] Long-term Follow-up (LTFU) of Onasemnogene Abeparvovec Gene Therapy in Spinal Muscular Atrophy (SMA)
    Day, J.
    Mendell, J.
    Finkel, R.
    Mercuri, E.
    Strauss, K.
    Kleyn, A.
    Chand, D.
    Tauscher-Wisniewski, S.
    Meriggioli, M.
    ANNALS OF NEUROLOGY, 2021, 90 : S151 - S152
  • [50] Expanding the Availability of Onasemnogene Abeparvovec to Older Patients: The Evolving Treatment Landscape for Spinal Muscular Atrophy
    Rene, Charlotte A.
    Parks, Robin J.
    PHARMACEUTICS, 2023, 15 (06)