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Taliglucerase alfa in Gaucher disease: Description of a Brazilian experience.
Cravo, R; Rotman, V; Oliveira, P M N; Defendi, H G T; Conceição, D A; Xavier, J R; Chertkoff, R; Noronha, T G; Maia, M L S.
Affiliation
  • Cravo R; Hemorio Hospital, Rio de Janeiro, Brazil. Electronic address: renata.cravo@hemorio.rj.gov.br.
  • Rotman V; BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: vrotman@gmail.com.
  • Oliveira PMN; BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: Patricia.mouta@bio.fiocruz.br.
  • Defendi HGT; BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: Hugo.defendi@bio.fiocruz.br.
  • Conceição DA; BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: Daraujo@bio.fiocruz.br.
  • Xavier JR; BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: janaina.xavier@bio.fiocruz.br.
  • Chertkoff R; Protalix Biotherapeutics, Carmiel, Israel. Electronic address: raul@protalix.com.
  • Noronha TG; BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: Tnoronha@bio.fiocruz.br.
  • Maia MLS; BIO-Manguinhos/Oswaldo Cruz Institute, Rio de Janeiro, Brazil. Electronic address: lourdes.maia@bio.fiocruz.br.
Blood Cells Mol Dis ; 68: 160-162, 2018 02.
Article in En | MEDLINE | ID: mdl-28131618
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.
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Enzyme Replacement Therapy / Gaucher Disease / Glucosylceramidase Type of study: Observational_studies / Risk_factors_studies Limits: Adult / Aged / Female / Humans / Male / Middle aged Country/Region as subject: America do sul / Brasil Language: En Journal: Blood Cells Mol Dis Journal subject: HEMATOLOGIA Year: 2018 Type: Article
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Enzyme Replacement Therapy / Gaucher Disease / Glucosylceramidase Type of study: Observational_studies / Risk_factors_studies Limits: Adult / Aged / Female / Humans / Male / Middle aged Country/Region as subject: America do sul / Brasil Language: En Journal: Blood Cells Mol Dis Journal subject: HEMATOLOGIA Year: 2018 Type: Article