Taliglucerase alfa in Gaucher disease: Description of a Brazilian experience

dc.creatorCravo, Renata
dc.creatorRotman, Vivian
dc.creatorOliveira, Patricia Mouta
dc.creatorDefendi, Hugo
dc.creatorConceição, D. Araujo
dc.creatorXavier, Janaina Xavier
dc.creatorChertkoff, Raul
dc.creatorNoronha, Tatiana Guimarães
dc.creatorMaia, Maria De Lourdes Sousa
dc.date2019-04-10T17:15:09Z
dc.date2019-04-10T17:15:09Z
dc.date2018
dc.date.accessioned2023-09-27T00:12:50Z
dc.date.available2023-09-27T00:12:50Z
dc.identifierCRAVO, Renata et al. Taliglucerase alfa in Gaucher disease: Description of a Brazilian experience. Blood Cells, Molecules & Diseases, La Jolla, v. 68, p. 160-162, 2018.
dc.identifier1096-0961
dc.identifierhttps://www.arca.fiocruz.br/handle/icict/32443
dc.identifier10.1016/j.bcmd.2017.01.005
dc.identifier.urihttps://repositorioslatinoamericanos.uchile.cl/handle/2250/8898576
dc.descriptionWe evaluated retrospectively, efficacy and safety of taliglucerase alfa for Gaucher disease in a Brazilian population. Thirteen patients were included for efficacy analysis only one of them naïve to enzyme replacement therapy. All the parameters evaluated remained stable throughout treatment (mean duration 3,5 years). Only three patients (out of 35) had to discontinue treatment due to a serious adverse event. In conclusion, treatment with taliglucerase alfa was found to be safe and efficient.
dc.formatapplication/pdf
dc.languageeng
dc.rightsrestricted access
dc.subjectGaucher disease
dc.subjectLysosomal storage diseases
dc.subjectEnzyme replacement therapy
dc.subjectTaliglucerase alfa
dc.subjectImiglucerase
dc.subjectVelaglucerase
dc.titleTaliglucerase alfa in Gaucher disease: Description of a Brazilian experience
dc.typeArticle


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