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Development and validation of Gaucher disease type 1 (GD1)-..:
Elstein, Deborah
;
Belmatoug, Nadia
;
Deegan, Patrick
...
https://www.repository.cam.ac.uk/handle/1810/333775. , 2022
Link:
https://www.repository.cam.ac.uk/handle/1810/333775
RT Journal T1
Development and validation of Gaucher disease type 1 (GD1)-specific patient-reported outcome measures (PROMs) for clinical monitoring and for clinical trials
UL https://suche.suub.uni-bremen.de/peid=base-ftunivcam:oai:www.repository.cam.ac.uk:1810_333775&Exemplar=1&LAN=DE A1 Elstein, Deborah A1 Belmatoug, Nadia A1 Deegan, Patrick A1 Göker-Alpan, Özlem A1 Hughes, Derralynn A A1 Schwartz, Ida Vanessa D A1 Weinreb, Neal A1 Bonner, Nicola A1 Panter, Charlotte A1 Fountain, Donna A1 Lenny, Andrew A1 Longworth, Louise A1 Miller, Rachael A1 Shah, Koonal A1 Schenk, Jörn A1 Sen, Rohini A1 Zimran, Ari PB Springer Science and Business Media LLC; http://dx.doi.org/10.1186/s13023-021-02163-y; Orphanet J Rare Dis YR 2022 K1 Content validation K1 Gaucher disease K1 Lysosomal storage disorder K1 PROM K1 Patient-reported outcomes K1 Psychometric validation K1 Questionnaire K1 Adult K1 Humans K1 Patient Reported Outcome Measures K1 Psychometrics K1 Quality of Life K1 Reproducibility of Results K1 Surveys and Questionnaires JF https://www.repository.cam.ac.uk/handle/1810/333775 LK http://dx.doi.org/https://www.repository.cam.ac.uk/handle/1810/333775 DO https://www.repository.cam.ac.uk/handle/1810/333775 SF ELIB - SuUB Bremen
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