Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials

被引：0
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作者：
Deborah Elstein
Nadia Belmatoug
Patrick Deegan
Özlem Göker-Alpan
Derralynn A. Hughes
Ida Vanessa D. Schwartz
Neal Weinreb
Nicola Bonner
Charlotte Panter
Donna Fountain
Andrew Lenny
Louise Longworth
Rachael Miller
Koonal Shah
Jörn Schenk
Rohini Sen
Ari Zimran
机构：
[1] Takeda Pharmaceuticals International AG,Assistance
[2] Université de Paris,Publique Hôpitaux de Paris Nord
[3] Cambridge University Hospitals,Lysosomal Disorders Unit
[4] O&O Alpan LLC,Lysosomal Disorders Unit and Center for Clinical Trials
[5] Royal Free London NHS Foundation Trust,Medical Genetics Service
[6] UFRGS, HCPA, Genetics Department
[7] University of Miami Miller School of Medicine,Gaucher Unit
[8] Adelphi Values,undefined
[9] PHMR,undefined
[10] Takeda Pharmaceuticals Company Ltd,undefined
[11] Shaare Zedek Medical Center,undefined
[12] Hebrew University-Hadassah Medical School,undefined
来源：
Orphanet Journal of Rare Diseases | / 17卷
关键词：
Patient-reported outcomes; PROM; Gaucher disease; Lysosomal storage disorder; Questionnaire; Content validation; Psychometric validation;
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