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Combined miglustat and enzyme replacement therapy in two patients with type 1 Gaucher disease: two case reports
BACKGROUND: Intravenous enzyme replacement therapy is a first-line therapy for Gaucher disease type 1, and substrate reduction therapy represents an oral treatment alternative. Both enzyme replacement therapy and substrate reduction therapy are generally used as monotherapies in Gaucher disease. How...
Autores principales: | , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
BioMed Central
2018
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Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5787317/ https://www.ncbi.nlm.nih.gov/pubmed/29373994 http://dx.doi.org/10.1186/s13256-017-1541-7 |