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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Pubblicato in:J Med Case Rep
Autori principali: Giuffrida, Gaetano, Lombardo, Rita, Di Francesco, Ernesto, Parrinello, Laura, Di Raimondo, Francesco, Fiumara, Agata
Natura: Artigo
Lingua:Inglês
Pubblicazione: BioMed Central 2016
Soggetti:
Case Report
Accesso online: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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