Treatment expectations and patient-reported outcomes of nusinersen therapy in adult spinal muscular atrophy

被引：0
作者：
Alma Osmanovic
Gresa Ranxha
Mareike Kumpe
Lars Müschen
Camilla Binz
Flavia Wiehler
Lejla Paracka
Sonja Körner
Katja Kollewe
Susanne Petri
Olivia Schreiber-Katz
机构：
[1] Hannover Medical School,Department of Neurology
来源：
Journal of Neurology | 2020年 / 267卷
关键词：
Spinal muscular atrophy (SMA); Patient-reported outcomes (PROs); Stanford expectations of treatment scale (SETS); Nusinersen; Antisense-oligonucleotide (ASO);
D O I：
暂无
中图分类号：
学科分类号：
摘要：
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页码：2398 / 2407
页数：9
相关论文
共 467 条
  • [1] Hoy SM(2017)Nusinersen: first global approval Drugs 77 473-479
  • [2] D'Amico A(2011)Spinal muscular atrophy Orphanet J Rare Dis 6 71-846
  • [3] Mercuri E(2015)Spinal muscular atrophy Neurol Clin 33 831-712
  • [4] Tiziano FD(2005)Natural history of denervation in SMA: relation to age, SMN2 copy number, and function Ann Neurol 57 704-165
  • [5] Bertini E(1995)Identification and characterization of a spinal muscular atrophy-determining gene Cell 80 155-1732
  • [6] Kolb SJ(2012)Evaluation of SMN protein, transcript, and copy number in the biomarkers for spinal muscular atrophy (BforSMA) clinical study PLoS ONE 7 e33572-635
  • [7] Kissel JT(2017)Nusinersen versus Sham Control in infantile-onset spinal muscular atrophy N Engl J Med 377 1723-105
  • [8] Swoboda KJ(2018)Nusinersen versus Sham control in later-onset spinal muscular atrophy N Engl J Med 378 625-3026
  • [9] Prior TW(2017)Pharmacology of antisense drugs Annu Rev Pharmacol Toxicol 57 81-325
  • [10] Scott CB(2016)Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study Lancet 388 3017-271
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