Long-term comparative efficacy and safety of risdiplam versus nusinersen in children with Type 1 spinal muscular atrophy (SMA)

被引:1
|
作者
Sajeev, G. [1 ]
Evans, R. [2 ]
Hawkins, N. [2 ,3 ]
Mahajan, A. [4 ]
Scott, D. [2 ]
Nam, J. [5 ]
Sutherland, S. [5 ]
Kokaliaris, C. [5 ,6 ]
机构
[1] Anal Grp Inc, Boston, MA USA
[2] Visible Analyt, Oxford, England
[3] Univ Glasgow, Inst Hlth & Wellbeing, Glasgow, Lanark, Scotland
[4] Bridge Med Consulting Ltd, London, England
[5] F Hoffmann La Roche Ltd, Global Access, Basel, Switzerland
[6] Roche Prod Ltd, Welwyn Garden City, Herts, England
关键词
D O I
10.1016/j.nmd.2023.07.385
中图分类号
R74 [神经病学与精神病学];
学科分类号
摘要
P08
引用
收藏
页码:S164 / S164
页数:1
相关论文
共 50 条
  • [21] Risdiplam in Type 1 Spinal Muscular Atrophy
    Krandick, Guido
    MONATSSCHRIFT KINDERHEILKUNDE, 2021, 169 (11) : 995 - 997
  • [22] Long-term follow-up study in patients with spinal muscular atrophy (SMA) receiving risdiplam treatment
    Tanvir, I
    Tam, S.
    O'Donnell, D.
    Shapouri, S.
    Lim, E.
    Shah, R.
    Sun, C.
    Seth, G.
    ANNALS OF NEUROLOGY, 2023, 94 : S119 - S119
  • [23] Long-Term Effects of Nusinersen Combined Physiotherapy in Spinal Muscular Atrophy Type 1: A Case Study
    Yagcioglu, Gullu Aydin
    Bulut, Numan
    Ugur, Fatma
    Gurbuz, Ipek Alemdaroglu
    Karaduman, A. Ayse
    Yilmaz, Oznur
    JOURNAL OF DR BEHCET UZ CHILDRENS HOSPITAL, 2022, 12 (01): : 91 - 96
  • [24] Long-term benefits of nusinersen in a child affected by cystic fibrosis and spinal muscular atrophy type 1
    Bruno, Giorgia
    Donnarumma, Bernadette
    Inverardi, Alessia
    Buonpensiero, Paolo
    Sepe, Angela
    Tosco, Antonella
    Raia, Valeria
    Varone, Antonio
    PEDIATRIC PULMONOLOGY, 2021, 56 (06) : 1806 - 1808
  • [25] Safety update: Risdiplam clinical trial program for spinal muscular atrophy (SMA)
    Baranello, G.
    Chiriboga, C.
    Servais, L.
    Darras, B.
    Day, J.
    Deconinck, N.
    Farrar, M.
    Finkel, R.
    Bertini, E.
    Kirschner, J.
    Rasson, M.
    Mazurkiewicz-Beldzinska, M.
    Vlodavets, D.
    Bader-Weder, S.
    Gorni, K.
    Jaber, B.
    Yeung, W.
    Papp, G.
    Scalco, R.
    Mercuri, E.
    NEUROMUSCULAR DISORDERS, 2023, 33 : S92 - S93
  • [26] Common complications in spinal muscular atrophy (SMA) type 1 after nusinersen treatment
    Guzin, Yigithan
    Buyuksen, Osman
    Gencpinar, Pinar
    Dundar, Nihal Olgac
    Baydan, Figen
    TURKISH JOURNAL OF PEDIATRICS, 2024, 66 (05) : 567 - 577
  • [27] Correction to: Two‑year efficacy and safety of risdiplam in patients with type 2 or non‑ambulant type 3 spinal muscular atrophy (SMA)
    Maryam Oskoui
    John W. Day
    Nicolas Deconinck
    Elena S. Mazzone
    Andres Nascimento
    Kayoko Saito
    Carole Vuillerot
    Giovanni Baranello
    Nathalie Goemans
    Janbernd Kirschner
    Anna Kostera-Pruszczyk
    Laurent Servais
    Gergely Papp
    Ksenija Gorni
    Heidemarie Kletzl
    Carmen Martin
    Tammy McIver
    Renata S. Scalco
    Hannah Staunton
    Wai Yin Yeung
    Paulo Fontoura
    Eugenio Mercuri
    Journal of Neurology, 2023, 270 : 2547 - 2549
  • [28] Safety and efficacy of nusinersen in spinal muscular atrophy: The EMBRACE study
    Acsadi, Gyula
    Crawford, Thomas O.
    Mueller-Felber, Wolfgang
    Shieh, Perry B.
    Richardson, Randal
    Natarajan, Niranjana
    Castro, Diana
    Ramirez-Schrempp, Daniela
    Gambino, Giulia
    Sun, Peng
    Farwell, Wildon
    MUSCLE & NERVE, 2021, 63 (05) : 668 - 677
  • [29] Safety and Efficacy of Nusinersen and Risdiplam for Spinal Muscular Atrophy: A Systematic Review and Meta-Analysis of Randomized Controlled Trials
    Qiao, Yue
    Chi, Yuewei
    Gu, Jian
    Ma, Ying
    BRAIN SCIENCES, 2023, 13 (10)
  • [30] RAINBOWFISH: Preliminary efficacy and safety data in risdiplam-treated infants with presymptomatic spinal muscular atrophy (SMA)
    Finkel, R.
    Farrar, M.
    Vlodavets, D.
    Zanoteli, E.
    Al-Muhaizea, M.
    Nelson, L.
    Prufer, A.
    Servais, L.
    Wang, Y.
    Fisher, C.
    Gerber, M.
    Gorni, K.
    Kletzl, H.
    Palfreeman, L.
    Scalco, R.
    Bertini, E.
    NEUROMUSCULAR DISORDERS, 2022, 32 : S85 - S86