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Improvement of life quality measured by Lansky Score after enzymatic replacement therapy in children with Gaucher disease type 1
BACKGROUND: Gaucher disease type 1 (GD1, OMIM# 230800), is a condition with high impact in patient's quality of life (QoL). We report the improvement in QoL of children with GD1 measured by Lansky play‐performance scale (LS) after enzymatic replacement therapy (ERT) and to describe our experien...
Autores principales: | , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
John Wiley and Sons Inc.
2017
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5823673/ https://www.ncbi.nlm.nih.gov/pubmed/29471591 http://dx.doi.org/10.1002/mgg3.339 |