title: Taliglucerase alfa in Gaucher disease: Description of a Brazilian experience.
abstract: We 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 3,5years). 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.
authors.affiliation: Hemorio Hospital, Rio de Janeiro, Brazil. Electronic address: renata.cravo@hemorio.rj.gov.br.
authors.forename: R
authors.initials: R
authors.lastname: Cravo
authors.affiliation: BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: vrotman@gmail.com.
authors.forename: V
authors.initials: V
authors.lastname: Rotman
authors.affiliation: BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: Patricia.mouta@bio.fiocruz.br.
authors.forename: P M N
authors.initials: PMN
authors.lastname: Oliveira
authors.affiliation: BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: Hugo.defendi@bio.fiocruz.br.
authors.forename: H G T
authors.initials: HGT
authors.lastname: Defendi
authors.affiliation: BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: Daraujo@bio.fiocruz.br.
authors.forename: D A
authors.initials: DA
authors.lastname: Conceição
authors.affiliation: BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: janaina.xavier@bio.fiocruz.br.
authors.forename: J R
authors.initials: JR
authors.lastname: Xavier
authors.affiliation: Protalix Biotherapeutics, Carmiel, Israel. Electronic address: raul@protalix.com.
authors.forename: R
authors.initials: R
authors.lastname: Chertkoff
authors.affiliation: BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: Tnoronha@bio.fiocruz.br.
authors.forename: T G
authors.initials: TG
authors.lastname: Noronha
authors.affiliation: BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: lourdes.maia@bio.fiocruz.br.
authors.forename: M L S
authors.initials: MLS
authors.lastname: Maia
mesh_terms: D000328:Adult; D000368:Aged; D001938:Brazil / Q000453:epidemiology; D056947:Enzyme Replacement Therapy / Q000379:methods*; D005260:Female; D005776:Gaucher Disease / Q000188:drug therapy* / Q000453:epidemiology; D005962:Glucosylceramidase / Q000009:adverse effects / Q000627:therapeutic use*; D006801:Humans; D008297:Male; D008875:Middle Aged; D012189:Retrospective Studies; D016896:Treatment Outcome
chemical_list: D005962:Glucosylceramidase; C582473:taliglucerase alfa
keywords: Enzyme replacement therapy; Gaucher disease; Imiglucerase; Lysosomal storage diseases; Taliglucerase alfa; Velaglucerase