Patient reported outcome measures in a large cohort of patients with type 1 Gaucher disease

BACKGROUND: It is now acknowledged that the input of patients in health outcome assessment is vital to understanding the impact of diseases and interventions for those diseases. This study is the first report of patient-reported outcome measures (PROM) in a large cohort of patients with type 1 Gauch...

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Опубликовано в: :Orphanet J Rare Dis
Главные авторы: Dinur, Tama, Istaiti, Majdolen, Frydman, Dafna, Becker-Cohen, Michal, Szer, Jeff, Zimran, Ari, Revel-Vilk, Shoshana
Формат: Artigo
Язык:Inglês
Опубликовано: BioMed Central 2020
Предметы:
Research
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC7556970/
https://ncbi.nlm.nih.gov/pubmed/33050940
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-020-01544-z
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