Assessment of Treatment Effect With Multiple Outcomes in 2 Clinical Trials of Patients With Duchenne Muscular Dystrophy.

Daniel Li,Craig M McDonald,Gary L Elfring,Marcio Souza,Joseph McIntosh,Dae Hyun Kim,Lee-Jen Wei

JAMA NETWORK OPEN（2020）

引用 9|浏览29

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摘要

This cohort study applies a heuristic, analytical procedure using z scores to data from 2 independent trials with multiple end points that examined whether ataluren is beneficial vs placebo for treating patients with Duchenne muscular dystrophy.

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