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Updated: Aug 15, 2026

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用PROPEL 3期数据验证患者报告结果测量信息系统 (PROMIS®) 物理功能问卷在晚发佩病的验证

Priya S Kishnani1, Simon Shohet2, Syed Raza3

  • 1Duke University, 905 Lasalle Street, GSRB1, Room 4010, Durham, NC, 27710, USA.

Journal of patient-reported outcomes
|January 31, 2024
PubMed
概括

患者报告结果测量信息系统 (PROMIS) 身体功能简短表格20a (PF20a) 有效测量晚发佩病 (LOPD) 的身体功能. 这种经过验证的工具有助于跟踪接受酶替代疗法 (ERT) 的LOPD患者的疾病进展和治疗反应.

关键词:
晚期发病的庞贝病 晚期发病的庞贝病普罗佩尔 (Propel) 是一个子.患者报告结果测量信息系统 (PROMIS)患者报告的结果物理功能 物理功能生活质量生活的质量.验证 验证 验证 验证

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