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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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Podrobná bibliografie
Vydáno v:Orphanet J Rare Dis
Hlavní autoři: Dinur, Tama, Istaiti, Majdolen, Frydman, Dafna, Becker-Cohen, Michal, Szer, Jeff, Zimran, Ari, Revel-Vilk, Shoshana
Médium: Artigo
Jazyk:Inglês
Vydáno: BioMed Central 2020
Témata:
On-line přístup: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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