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Position Statement: Sharing of Clinical Research Data in Spinal Muscular Atrophy to Accelerate Research and Improve Outcomes for Patients.

作者信息

Lochmüller Hanns, Evans David, Farwell Wildon, Finkel Richard, Goemans Nathalie, de Lemus Mencia, Matyushenko Vitaliy, Muntoni Francesco, Ouillade Marie-Christine, Schwersenz Inge, Wilson Petra

机构信息

Department of Neuropediatrics and Muscle Disorders, Medical Center - University ofFreiburg, Faculty of Medicine, Freiburg, Germany.

Data Science - Personalized HealthCare, F. Hoffmann-La Roche, Basel, Switzerland.

出版信息

J Neuromuscul Dis. 2018;5(2):131-133. doi: 10.3233/JND-180325.

DOI:10.3233/JND-180325
PMID:29865093
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC6004907/
Abstract
摘要

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本文引用的文献

1
Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy.依库珠单抗治疗晚发性脊髓性肌萎缩症的疗效观察：一项随机、双盲、安慰剂对照 3 期试验
N Engl J Med. 2018 Feb 15;378(7):625-635. doi: 10.1056/NEJMoa1710504.
2
Advances in therapy for spinal muscular atrophy: promises and challenges.脊髓性肌萎缩症治疗的进展：前景与挑战。
Nat Rev Neurol. 2018 Apr;14(4):214-224. doi: 10.1038/nrneurol.2018.4. Epub 2018 Feb 9.
3
Natural History, Trial Readiness and Gene Discovery: Advances in Patient Registries for Neuromuscular Disease.自然病史、临床试验准备和基因发现：神经肌肉疾病患者登记研究的进展。
Adv Exp Med Biol. 2017;1031:97-124. doi: 10.1007/978-3-319-67144-4_5.
4
The International Rare Diseases Research Consortium: Policies and Guidelines to maximize impact.国际罕见病研究联合会：最大化影响的政策与指南。
Eur J Hum Genet. 2017 Dec;25(12):1293-1302. doi: 10.1038/s41431-017-0008-z. Epub 2017 Nov 20.
5
Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy.依库珠单抗治疗婴儿型脊髓性肌萎缩症的疗效观察
N Engl J Med. 2017 Nov 2;377(18):1723-1732. doi: 10.1056/NEJMoa1702752.
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