Opportunities for improving therapy development in ALS

被引：9

作者：

Bruijn, Lucie ^{[1
]}

Cudkowicz, Merit ^{[2
]}

机构：

[1] ALS Assoc, Washington, DC 20005 USA

[2] Massachusetts Gen Hosp, Neurol Clin Res Inst, Boston, MA 02114 USA

来源：

AMYOTROPHIC LATERAL SCLEROSIS AND FRONTOTEMPORAL DEGENERATION | 2014年 / 15卷 / 3-4期

关键词：

Amyotrophic lateral sclerosis; clinical trials; biomarkers; therapy; AMYOTROPHIC-LATERAL-SCLEROSIS; RANDOMIZED SEQUENTIAL TRIAL; PHASE-III; MULTICENTER; SURVIVAL; LITHIUM; DESIGNS;

D O I：

10.3109/21678421.2013.872662

中图分类号：

R74 [神经病学与精神病学];

学科分类号：

摘要：

In May 2013, The ALS Association and The Northeast ALS Consortium (NEALS) convened a meeting of stakeholders for a round-table discussion of ways to improve therapy development in ALS. The following overview summarizes issues raised and potential new directions discussed at the meeting. We recommend that future phase II clinical trials in ALS proceed when the proposed treatment is directed at targets that are likely to be involved in ALS pathogenesis in a defined subgroup of patients, and be accompanied by one or more biomarkers to track both clinical progression and pharmacodynamic engagement of the target. Innovations in trial structure and design, and greater involvement of patient advocates, may also improve trials.

引用

页码：169 / 173

页数：5

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