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

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;

D O I：

暂无

中图分类号：

学科分类号：

摘要：

引用

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