Development of the Fabry Disease Patient-Reported Outcome (FD-PRO): a new instrument to measure the symptoms and impacts of Fabry Disease

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作者
Alaa Hamed
Pronabesh DasMahapatra
Nicole Lyn
Chad Gwaltney
Robert J. Hopkin
机构
[1] Sanofi Genzyme,
[2] Gwaltney Consulting,undefined
[3] Cincinnati Children’s Hospital Medical Center and University of Cincinnati College of Medicine Department of Pediatrics,undefined
关键词
Fabry disease; Patient-reported outcome; FD-PRO; Clinical outcome assessment; Rare disease; Patient experience; Qualitative interviews;
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