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Updated: Jul 18, 2025

In Vitro Enzyme Measurement to Test Pharmacological Chaperone Responsiveness in Fabry and Pompe Disease
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患者报告的Gaucher病结果:系统性审查

Junchao Feng1,2,3, Zhongchun Gao4, Zhao Shi1,2,3

  • 1Centre for Health Management and Policy Research, School of Public Health, Cheeloo College of Medicine, Shandong University, Jinan, 250012, China.

Orphanet journal of rare diseases
|August 25, 2023
PubMed
概括

患者报告的结果 (PROs) 对高氏病 (GD) 管理至关重要. 本次审查强调需要更好的PRO仪器来准确测量GD患者的生活质量和治疗疗效.

关键词:
氏病是高希氏病的一种疾病.文献综述 文献综述 文献综述患者报告的结果.生活质量生活的质量.罕见疾病是一种罕见的疾病.

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A Quick Phenotypic Neurological Scoring System for Evaluating Disease Progression in the SOD1-G93A Mouse Model of ALS06:49

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