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Lessons from the HEALEY adaptive platform trial in amyotrophic lateral sclerosis.

作者信息

Paganoni Sabrina, Harkey Brittney, Giacomelli Elisa, Cudkowicz Merit

机构信息

Sean M. Healey and AMG Center for ALS & the Neurological Clinical Research Institute, Massachusetts General Hospital, Harvard Medical School, Boston, MA, USA.

Spaulding Rehabilitation Hospital, Harvard Medical School, Boston, MA, USA.

出版信息

Nat Aging. 2024 Nov;4(11):1512-1515. doi: 10.1038/s43587-024-00740-x.

DOI:10.1038/s43587-024-00740-x
PMID:39487328
Abstract
摘要

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2
Protocol for a randomized, placebo-controlled, double-blind phase IIa study of the safety, tolerability, and symptomatic efficacy of the ROCK-inhibitor Fasudil in patients with Parkinson's disease (ROCK-PD).一项关于ROCK抑制剂法舒地尔在帕金森病患者中的安全性、耐受性和症状疗效的随机、安慰剂对照、双盲IIa期研究方案（ROCK-PD）
Front Aging Neurosci. 2024 Feb 14;16:1308577. doi: 10.3389/fnagi.2024.1308577. eCollection 2024.
3
Biomarkers in amyotrophic lateral sclerosis: current status and future prospects.
肌萎缩侧索硬化症中的生物标志物：现状与未来展望。
Nat Rev Neurol. 2023 Dec;19(12):754-768. doi: 10.1038/s41582-023-00891-2. Epub 2023 Nov 10.
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Exploring Advancements in the Treatment of Amyotrophic Lateral Sclerosis: A Comprehensive Review of Current Modalities and Future Prospects.探索肌萎缩侧索硬化症治疗的进展：当前治疗方式及未来前景的全面综述
Cureus. 2023 Sep 18;15(9):e45489. doi: 10.7759/cureus.45489. eCollection 2023 Sep.
5
Design and Statistical Innovations in a Platform Trial for Amyotrophic Lateral Sclerosis.肌萎缩侧索硬化症平台试验的设计与统计创新
Ann Neurol. 2023 Sep;94(3):547-560. doi: 10.1002/ana.26714. Epub 2023 Jun 22.
6
Amyotrophic lateral sclerosis: translating genetic discoveries into therapies.肌萎缩侧索硬化症：将遗传发现转化为治疗方法。
Nat Rev Genet. 2023 Sep;24(9):642-658. doi: 10.1038/s41576-023-00592-y. Epub 2023 Apr 6.
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Lancet. 2022 Oct 15;400(10360):1363-1380. doi: 10.1016/S0140-6736(22)01272-7. Epub 2022 Sep 15.
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Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development.适应性平台试验改变肌萎缩侧索硬化症治疗开发。
Ann Neurol. 2022 Feb;91(2):165-175. doi: 10.1002/ana.26285. Epub 2022 Jan 10.
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