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Successful switch from enzyme replacement therapy to miglustat in an adult patient with type 1 Gaucher disease: a case report
BACKGROUND: Gaucher disease is one of the most common lipid-storage disorders, affecting approximately 1 in 75,000 births. Enzyme replacement therapy with recombinant glucocerebrosidase is currently considered the first-line treatment choice for patients with symptomatic Gaucher disease type 1. Oral...
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| Publicado no: | J Med Case Rep |
|---|---|
| Main Authors: | , , , , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
BioMed Central
2016
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC5100336/ https://ncbi.nlm.nih.gov/pubmed/27821156 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13256-016-1060-y |
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