Biochemical response to substrate reduction therapy versus enzyme replacement therapy in Gaucher disease type 1 patients

BACKGROUND: We retrospectively compared biochemical responses in type 1 Gaucher disease patients to treatment with glycosphingolipid synthesis inhibitors miglustat and eliglustat and ERT. METHODS: Seventeen GD1 patients were included (n = 6 eliglustat, (two switched from ERT), n = 9 miglustat (seven...

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Publicat a:Orphanet J Rare Dis
Autors principals: Smid, Bouwien E., Ferraz, Maria J., Verhoek, Marri, Mirzaian, Mina, Wisse, Patrick, Overkleeft, Herman S., Hollak, Carla E., Aerts, Johannes M.
Format: Artigo
Idioma:Inglês
Publicat: BioMed Central 2016
Matèries:
Research
Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC4806476/
https://ncbi.nlm.nih.gov/pubmed/27008851
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-016-0413-3
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