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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Bibliografiska uppgifter
I publikationen:Orphanet J Rare Dis
Huvudupphovsmän: Dinur, Tama, Istaiti, Majdolen, Frydman, Dafna, Becker-Cohen, Michal, Szer, Jeff, Zimran, Ari, Revel-Vilk, Shoshana
Materialtyp: Artigo
Språk:Inglês
Publicerad: BioMed Central 2020
Ämnen:
Research
Länkar: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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