The Facioscapulohumeral Muscular Dystrophy-Health Index: Development and evaluation of a disease-specific outcome measure.

Anika Varma,Jennifer Weinstein,Jamison Seabury,Spencer Rosero, Charlotte Engebrecht,Ellen Wagner,Christine Zizzi,Elizabeth A Luebbe,Nuran Dilek,Michael P McDermott,John Kissel,Valeria Sansone,Chad Heatwole

Muscle & nerve（2023）

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摘要

The FSHD-HI provides researchers and clinicians with a reliable and valid mechanism to measure multifaceted disease burden in patients with FSHD. The FSHD-HI may facilitate quantification of therapeutic effectiveness, as demonstrated by its use as a secondary and exploratory measure in several clinical trials.

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关键词

clinical outcome assessment,clinical trial,drug label,facioscapulohumeral muscular dystrophy,patient-reported outcome

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