COST EFFECTIVENESS OF ONASEMNOGENE ABEPARVOVEC IN INFANTS WITH PRESYMPTOMATIC SPINAL MUSCULAR ATROPHY IN ITALY

被引：0
|
作者：
Valentini, I [1 ,2 ]
Ghetti, G. [3 ]
Pane, M. [4 ]
Cicchetti, A. [1 ,2 ]
Rumi, F. [5 ]
Di Brino, E. [1 ,2 ]
Basile, M. [5 ]
Pistillo, G. [6 ]
Bischof, M. [7 ]
机构：
[1] Cattolica Sacro Cuore Univ UCSC, Fac Econ, Rome, Italy
[2] Cattolica Sacro Cuore Univ, Grad Sch Hlth Econ & Management ALTEMS, Rome, Italy
[3] AdRes HEOR, Turin, To, Italy
[4] Fdn Policlin Univ Agostino Gemelli, Rome, Italy
[5] Univ Cattolica Sacro Cuore, Grad Sch Hlth Econ & Management ALTEMS, Rome, Italy
[6] Novartis Gene Therapies, Legnano, MI, Italy
[7] Novartis Gene Therapies GmbH, Rotkreuz, Switzerland
来源：
VALUE IN HEALTH | 2023年 / 26卷 / 12期
关键词：
D O I：
暂无
中图分类号：
F [经济];
学科分类号：
02 ;
摘要：
RWD129
引用
收藏
页码：S528 / S529
页数：2
相关论文
共 50 条
  • [21] Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
    Kevin A. Strauss
    Michelle A. Farrar
    Francesco Muntoni
    Kayoko Saito
    Jerry R. Mendell
    Laurent Servais
    Hugh J. McMillan
    Richard S. Finkel
    Kathryn J. Swoboda
    Jennifer M. Kwon
    Craig M. Zaidman
    Claudia A. Chiriboga
    Susan T. Iannaccone
    Jena M. Krueger
    Julie A. Parsons
    Perry B. Shieh
    Sarah Kavanagh
    Melissa Wigderson
    Sitra Tauscher-Wisniewski
    Bryan E. McGill
    Thomas A. Macek
    Nature Medicine, 2022, 28 : 1390 - 1397
  • [22] Onasemnogene Abeparvovec in Presymptomatic Spinal Muscular Atrophy (SMA): SPRINT Study Update in Children with Three Copies of SMN2
    Strauss, Kevin
    Muntoni, Francesco
    Farrar, Michelle
    Saito, Kayoko
    Mendell, Jerry
    Servais, Laurent
    McMillan, Hugh
    Finkel, Richard
    Swoboda, Kathryn
    Kwon, Jennifer
    Zaidman, Craig
    Chiriboga, Claudia
    Lannaccone, Susan
    Krueger, Jena
    Parsons, Julie
    Shieh, Perry
    Kavanagh, Sarah
    Chand, Deepa
    Tauscher-Wisniewski, Sitra
    McGill, Bryan
    Macek, Thomas
    NEUROLOGY, 2022, 98 (18)
  • [23] Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial
    Strauss, Kevin A.
    Farrar, Michelle A.
    Muntoni, Francesco
    Saito, Kayoko
    Mendell, Jerry R.
    Servais, Laurent
    McMillan, Hugh J.
    Finkel, Richard S.
    Swoboda, Kathryn J.
    Kwon, Jennifer M.
    Zaidman, Craig M.
    Chiriboga, Claudia A.
    Iannaccone, Susan T.
    Krueger, Jena M.
    Parsons, Julie A.
    Shieh, Perry B.
    Kavanagh, Sarah
    Wigderson, Melissa
    Tauscher-Wisniewski, Sitra
    McGill, Bryan E.
    Macek, Thomas A.
    NATURE MEDICINE, 2022, 28 (07) : 1390 - +
  • [24] Short-term clinical outcomes of onasemnogene abeparvovec treatment for spinal muscular atrophy
    Lee, Seungbok
    Lee, Yun Jeong
    Kong, Juhyun
    Ryu, Hye Won
    Shim, Young Kyu
    Han, Ji Yeon
    Woo, Hyewon
    Kim, Soo Yeon
    Cho, Anna
    Lim, Byung Chan
    Chae, Jong Hee
    BRAIN & DEVELOPMENT, 2022, 44 (04): : 287 - 293
  • [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] Cost comparison analysis of onasemnogene abeparvovec and nusinersen for treatment of patients with spinal muscular atrophy type 1 in the Netherlands
    van der Schans, Simon
    Velikanova, Rimma
    Weidlich, Diana
    Howells, Ruth
    Patel, Anish
    Bischof, Matthias
    Postma, Maarten J.
    Boersma, Cornelis
    EUROPEAN JOURNAL OF HEALTH ECONOMICS, 2025,
  • [27] Effect on maximal mouth opening in children with spinal muscular atrophy treated with onasemnogene abeparvovec
    Beri, Nidhi
    Kapoor, Lekha
    Parashar, Deepak
    Mundada, Vivek
    ARCHIVES OF DISEASE IN CHILDHOOD, 2023, 108 (10) : 866 - 867
  • [28] Onasemnogene Abeparvovec-xioi Gene Therapy in Presymptomatic Spinal Muscular Atrophy (SMA): SPR1NT Study Update
    Strauss, K. A.
    Farrar, M. A.
    Swoboda, K. J.
    Saito, K.
    Chiriboga, C. A.
    Finkel, R. S.
    Iannaccone, S. T.
    Krueger, J. M.
    Kwon, J. M.
    McMillan, H. J.
    Servais, L.
    Mendell, J. R.
    Parsons, J.
    Scoto, M.
    Shieh, P. B.
    Zaidman, C.
    Schultz, M.
    Baldinetti, F.
    Ogrinc, F. G.
    Kavanagh, S.
    Feltner, D. E.
    Tauscher-Wisniewski, S.
    McGill, B. E.
    Sproule, D. M.
    Muntoni, F.
    EUROPEAN JOURNAL OF NEUROLOGY, 2020, 27 : 165 - 166
  • [29] 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
  • [30] 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
12345