Meta-analysis of clinical impact of onasemnogene abeparvovec treatment of spinal muscular atrophy patients

被引：0
|
作者：
Reyna, S. [1 ]
Dabbous, O. [1 ]
Wallach, S. [1 ]
Patel, A. [1 ]
Toro, W. [1 ]
Ritter, S. [1 ]
机构：
[1] Novartis Gene Therapies Inc, Bannockburn, IL USA
来源：
NEUROMUSCULAR DISORDERS | 2024年 / 43卷
关键词：
D O I：
10.1016/j.nmd.2024.07.608
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
164P
引用
收藏
页数：1
相关论文
共 50 条
  • [21] COST EFFECTIVENESS OF ONASEMNOGENE ABEPARVOVEC IN INFANTS WITH PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY IN ITALY
    Valentini, I
    Ghetti, G.
    Pane, M.
    Cicchetti, A.
    Rumi, F.
    Di Brino, E.
    Basile, M.
    Pistillo, G.
    Bischof, M.
    VALUE IN HEALTH, 2023, 26 (12) : S528 - S529
  • [22] Epithelioid neoplasm of the spinal cord in a child with spinal muscular atrophy treated with onasemnogene abeparvovec
    Retson, Laura
    Tiwari, Nishant
    Vaughn, Jennifer
    Bernes, Saunder
    Adelson, P. David
    Mansfield, Keith
    Libertini, Silvana
    Kuzmiski, Brent
    Alecu, Iulian
    Gabriel, Richard
    Mangum, Ross
    MOLECULAR THERAPY, 2023, 31 (10) : 2991 - 2998
  • [23] Clinical Course in a Patient With Spinal Muscular Atrophy Type 0 Treated With Nusinersen and Onasemnogene Abeparvovec
    Matesanz, Susan E.
    Curry, Candace
    Gross, Brianna
    Rubin, Adam I.
    Linn, Rebecca
    Yum, Sabrina W.
    Kichula, Elizabeth A.
    JOURNAL OF CHILD NEUROLOGY, 2020, 35 (11) : 717 - 723
  • [24] 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
  • [25] Onasemnogene Abeparvovec Gene Therapy and Risdiplam for the Treatment of Spinal Muscular Atrophy in Thailand: A Cost-Utility Analysis
    Khuntha, Sarayuth
    Prawjaeng, Juthamas
    Ponragdee, Kunnatee
    Sanmaneechai, Oranee
    Srinonprasert, Varalak
    Leelahavarong, Pattara
    APPLIED HEALTH ECONOMICS AND HEALTH POLICY, 2025, 23 (02) : 277 - 290
  • [26] Patients with Spinal Muscular Atrophy Type 1 Achieve and Maintain Bulbar Function Following Onasemnogene Abeparvovec Treatment
    McGrattan, Katlyn E.
    Shell, Richard D.
    Hurst-Davis, Rebecca
    Young, Sally Dunaway
    O'Brien, Eamonn
    Lavrov, Arseniy
    Wallach, Shiri
    LaMarca, Nicole
    Reyna, Sandra P.
    Darras, Basil T.
    JOURNAL OF NEUROMUSCULAR DISEASES, 2023, 10 (04) : 531 - 540
  • [27] Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy
    Chand, Deepa
    Mohr, Franziska
    McMillan, Hugh
    Tukov, Francis Fonyuy
    Montgomery, Kyle
    Kleyn, Aaron
    Sun, Rui
    Tauscher-Wisniewski, Sitra
    Kaufmann, Petra
    Kullak-Ublick, Gerd
    JOURNAL OF HEPATOLOGY, 2021, 74 (03) : 560 - 566
  • [28] Outcomes in patients with spinal muscular atrophy given nusinersen, onasemnogene abeparvovec or no treatment: an analysis based on restricted mean survival time
    Bartoli, Laura
    Messori, Andrea
    EXPERT OPINION ON ORPHAN DRUGS, 2020, 8 (08): : 303 - 307
  • [29] Treatment of spinal muscular atrophy with Onasemnogene Abeparvovec in Switzerland: a prospective observational case series study
    Stettner, Georg M.
    Hasselmann, Oswald
    Tscherter, Anne
    Galiart, Elea
    Jacquier, David
    Klein, Andrea
    BMC NEUROLOGY, 2023, 23 (01)
  • [30] 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
12345