Systematic Literature Review of Clinical and Economic Evidence for Spinal Muscular Atrophy

被引：0
|
作者：
Min Yang
Hiroyuki Awano
Satoru Tanaka
Walter Toro
Su Zhang
Omar Dabbous
Ataru Igarashi
机构：
[1] Analysis Group,Department of Pediatrics
[2] Inc.,Unit of Public Health and Preventive Medicine
[3] Kobe University Graduate School of Medicine,Department of Health Economics and Outcomes Research, Graduate School of Pharmaceutical Sciences
[4] Novartis Pharma K.K.,undefined
[5] Novartis Gene Therapies,undefined
[6] Inc.,undefined
[7] Yokohama City University,undefined
[8] The University of Tokyo,undefined
来源：
Advances in Therapy | 2022年 / 39卷
关键词：
Disease-modifying therapies; Economic burden; Gene therapy; Health-related quality of life; Humanistic burden; Natural history; Nusinersen; Onasemnogene abeparvovec; Spinal muscular atrophy; Systematic literature review;
D O I：
暂无
中图分类号：
学科分类号：
摘要：
引用
收藏
页码：1915 / 1958
页数：43
相关论文
共 50 条
  • [11] Spinal Muscular Atrophy Scoliosis in the Era of Background Therapies-A Review of the Literature
    Ruythooren, Fred
    Moens, Pierre
    JOURNAL OF CLINICAL MEDICINE, 2024, 13 (12)
  • [12] Describing nutrition in spinal muscular atrophy: A systematic review
    Moore, Georgia E.
    Lindenmayer, Amara W.
    McConchie, Grace A.
    Ryan, Monique M.
    Davidson, Zoe E.
    NEUROMUSCULAR DISORDERS, 2016, 26 (07) : 395 - 404
  • [13] Systematic Review of Presymptomatic Treatment for Spinal Muscular Atrophy
    Cooper, Katy
    Nalbant, Gamze
    Sutton, Anthea
    Harnan, Sue
    Thokala, Praveen
    Chilcott, Jim
    McNeill, Alisdair
    Bessey, Alice
    INTERNATIONAL JOURNAL OF NEONATAL SCREENING, 2024, 10 (03)
  • [14] A Systematic Literature Review of the Natural History of Respiratory, Swallowing, Feeding, and Speech Functions in Spinal Muscular Atrophy (SMA)
    Marti, Yasmina
    Ribero, Valerie Aponte
    Batson, Sarah
    Mitchell, Stephen
    Gorni, Ksenija
    Gusset, Nicole
    Oskoui, Maryam
    Servais, Laurent
    Deconinck, Nicolas
    Mcgrattan, Katlyn Elizabeth
    Mercuri, Eugenio
    Sutherland, C. Simone
    JOURNAL OF NEUROMUSCULAR DISEASES, 2024, 11 (05) : 889 - 904
  • [15] The impact of three SMN2 gene copies on clinical characteristics and effect of disease-modifying treatment in patients with spinal muscular atrophy: a systematic literature review
    Dosi, Claudia
    Masson, Riccardo
    FRONTIERS IN NEUROLOGY, 2024, 15
  • [16] Cost-Effectiveness of Technologies for the Treatment of Spinal Muscular Atrophy: A Systematic Review of Economic Studies
    Motta-Santos, Andre
    Noronha, Kenya
    Reis, Carla
    Freitas, Daniela
    Carvalho, Lelia
    Andrade, Monica
    VALUE IN HEALTH REGIONAL ISSUES, 2024, 42
  • [17] Measuring quality of life in children with spinal muscular atrophy: a systematic literature review
    Vaidya, Shalvaree
    Boes, Stefan
    QUALITY OF LIFE RESEARCH, 2018, 27 (12) : 3087 - 3094
  • [18] Systematic Literature Review to Assess the Cost and Resource Use Associated with Spinal Muscular Atrophy Management
    Paracha, Noman
    Hudson, Pollyanna
    Mitchell, Stephen
    Sutherland, C. Simone
    PHARMACOECONOMICS, 2022, 40 (SUPPL 1) : 11 - 38
  • [19] Hydrocephalus in Spinal Muscular Atrophy: A Case Report and Review of the Literature
    Sah, Jeetendra P.
    Abrams, Aaron W.
    Chari, Geetha
    Linden, Craig
    Anziska, Yaacov
    JOURNAL OF PEDIATRIC NEUROLOGY, 2021, 19 (05) : 352 - 354
  • [20] Spinal muscular atrophy - clinical spectrum and therapy
    Walter, Maggie C.
    Stauber, Anne Julia
    FORTSCHRITTE DER NEUROLOGIE PSYCHIATRIE, 2018, 86 (09) : 543 - 550
12345