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Management goals of type 1 Gaucher disease in South Africa: An expert Delphi consensus document on good clinical practice
BACKGROUND: Gaucher disease is a rare autosomal recessive glycosphingolipid storage disease that ultimately leads to reduced life expectancy. Management of Gaucher disease is challenging due to its wide genotypic and phenotypic variability and changing clinical manifestations due to effective treatm...
| Autores principales: | , , |
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| Formato: | Online Artículo Texto |
| Lenguaje: | English |
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Public Library of Science
2023
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| Materias: | |
| Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10443848/ https://www.ncbi.nlm.nih.gov/pubmed/37607165 http://dx.doi.org/10.1371/journal.pone.0290401 |