Laronidase for treating mucopolysaccharidosis type I

Detalhes bibliográficos
Autor(a) principal: El Dib, Regina Paolucci [UNIFESP]
Data de Publicação: 2007
Outros Autores: Pastores, G. M.
Tipo de documento: Artigo
Idioma: eng
Título da fonte: Repositório Institucional da UNIFESP
Texto Completo: http://www.funpecrp.com.br/gmr/year2007/vol3-6/pdf/GMR0370.pdf
http://repositorio.unifesp.br/handle/11600/43719
Resumo: Mucopolysaccharidoses are a group of inherited metabolic diseases caused by the absence or deficiency of the lysosomal enzymes that are needed for breaking down glycosaminoglycans (GAGs). Over time, GAGs collect in cells, blood and connective tissues, and increased amounts are excreted in the urine. The result is permanent and includes progressive cell damage that affects the individual's appearance, physical abilities, organ and system functioning and, in certain cases, mental development. Enzyme replacement therapies are currently in use or are being tested for at least three different subtypes (I, II and VI). The aim of the present study was to evaluate the effectiveness and safety of laronidase for treating mucopolysaccharidosis type I. A systematic review of the literature was conducted. A computerized electronic search was then conducted using the CENTRAL, Pubmed, EMBASE, and LILACS databases, to identify any randomized controlled trials. The last date of the search was June 2006. There was no possibility of combining the results, because only one study was included. In the pivotal placebo-controlled trial conducted over a 26-week period, there was a reduction in the urinary excretion of GAGs among treated patients. Regarding adverse events, there were no laronidase-related serious adverse events or deaths. Laronidase seems to be a promising agent for treating mucopolysaccharidosis type I, as shown by the reduction in the urinary excretion of GAGs and the associated improvements in vital capacity and in the performance of defined physical tasks.
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spelling Laronidase for treating mucopolysaccharidosis type Imucopolysaccharidosis type 1Hurler syndromeiduronidaseMucopolysaccharidoses are a group of inherited metabolic diseases caused by the absence or deficiency of the lysosomal enzymes that are needed for breaking down glycosaminoglycans (GAGs). Over time, GAGs collect in cells, blood and connective tissues, and increased amounts are excreted in the urine. The result is permanent and includes progressive cell damage that affects the individual's appearance, physical abilities, organ and system functioning and, in certain cases, mental development. Enzyme replacement therapies are currently in use or are being tested for at least three different subtypes (I, II and VI). The aim of the present study was to evaluate the effectiveness and safety of laronidase for treating mucopolysaccharidosis type I. A systematic review of the literature was conducted. A computerized electronic search was then conducted using the CENTRAL, Pubmed, EMBASE, and LILACS databases, to identify any randomized controlled trials. The last date of the search was June 2006. There was no possibility of combining the results, because only one study was included. In the pivotal placebo-controlled trial conducted over a 26-week period, there was a reduction in the urinary excretion of GAGs among treated patients. Regarding adverse events, there were no laronidase-related serious adverse events or deaths. Laronidase seems to be a promising agent for treating mucopolysaccharidosis type I, as shown by the reduction in the urinary excretion of GAGs and the associated improvements in vital capacity and in the performance of defined physical tasks.Univ Fed Sao Paulo, Ctr Cochrane Brasil, Sao Paulo, SP, BrazilNYU, Sch Med, Neurogenet Lab, New York, NY USAUniv Fed Sao Paulo, Ctr Cochrane Brasil, Sao Paulo, SP, BrazilWeb of ScienceFunpec-editoraUniversidade Federal de São Paulo (UNIFESP)NYUEl Dib, Regina Paolucci [UNIFESP]Pastores, G. M.2018-06-15T17:30:20Z2018-06-15T17:30:20Z2007-01-01info:eu-repo/semantics/articleinfo:eu-repo/semantics/publishedVersion667-674application/pdfhttp://www.funpecrp.com.br/gmr/year2007/vol3-6/pdf/GMR0370.pdfGenetics And Molecular Research. Ribeirao Preto: Funpec-editora, v. 6, n. 3, p. 667-674, 2007.WOS000251696600023.pdf1676-5680http://repositorio.unifesp.br/handle/11600/43719WOS:000251696600023engGenetics And Molecular Researchinfo:eu-repo/semantics/openAccessreponame:Repositório Institucional da UNIFESPinstname:Universidade Federal de São Paulo (UNIFESP)instacron:UNIFESP2024-08-01T23:39:54Zoai:repositorio.unifesp.br/:11600/43719Repositório InstitucionalPUBhttp://www.repositorio.unifesp.br/oai/requestbiblioteca.csp@unifesp.bropendoar:34652024-08-01T23:39:54Repositório Institucional da UNIFESP - Universidade Federal de São Paulo (UNIFESP)false
dc.title.none.fl_str_mv Laronidase for treating mucopolysaccharidosis type I
title Laronidase for treating mucopolysaccharidosis type I
spellingShingle Laronidase for treating mucopolysaccharidosis type I
El Dib, Regina Paolucci [UNIFESP]
mucopolysaccharidosis type 1
Hurler syndrome
iduronidase
title_short Laronidase for treating mucopolysaccharidosis type I
title_full Laronidase for treating mucopolysaccharidosis type I
title_fullStr Laronidase for treating mucopolysaccharidosis type I
title_full_unstemmed Laronidase for treating mucopolysaccharidosis type I
title_sort Laronidase for treating mucopolysaccharidosis type I
author El Dib, Regina Paolucci [UNIFESP]
author_facet El Dib, Regina Paolucci [UNIFESP]
Pastores, G. M.
author_role author
author2 Pastores, G. M.
author2_role author
dc.contributor.none.fl_str_mv Universidade Federal de São Paulo (UNIFESP)
NYU
dc.contributor.author.fl_str_mv El Dib, Regina Paolucci [UNIFESP]
Pastores, G. M.
dc.subject.por.fl_str_mv mucopolysaccharidosis type 1
Hurler syndrome
iduronidase
topic mucopolysaccharidosis type 1
Hurler syndrome
iduronidase
description Mucopolysaccharidoses are a group of inherited metabolic diseases caused by the absence or deficiency of the lysosomal enzymes that are needed for breaking down glycosaminoglycans (GAGs). Over time, GAGs collect in cells, blood and connective tissues, and increased amounts are excreted in the urine. The result is permanent and includes progressive cell damage that affects the individual's appearance, physical abilities, organ and system functioning and, in certain cases, mental development. Enzyme replacement therapies are currently in use or are being tested for at least three different subtypes (I, II and VI). The aim of the present study was to evaluate the effectiveness and safety of laronidase for treating mucopolysaccharidosis type I. A systematic review of the literature was conducted. A computerized electronic search was then conducted using the CENTRAL, Pubmed, EMBASE, and LILACS databases, to identify any randomized controlled trials. The last date of the search was June 2006. There was no possibility of combining the results, because only one study was included. In the pivotal placebo-controlled trial conducted over a 26-week period, there was a reduction in the urinary excretion of GAGs among treated patients. Regarding adverse events, there were no laronidase-related serious adverse events or deaths. Laronidase seems to be a promising agent for treating mucopolysaccharidosis type I, as shown by the reduction in the urinary excretion of GAGs and the associated improvements in vital capacity and in the performance of defined physical tasks.
publishDate 2007
dc.date.none.fl_str_mv 2007-01-01
2018-06-15T17:30:20Z
2018-06-15T17:30:20Z
dc.type.driver.fl_str_mv info:eu-repo/semantics/article
dc.type.status.fl_str_mv info:eu-repo/semantics/publishedVersion
format article
status_str publishedVersion
dc.identifier.uri.fl_str_mv http://www.funpecrp.com.br/gmr/year2007/vol3-6/pdf/GMR0370.pdf
Genetics And Molecular Research. Ribeirao Preto: Funpec-editora, v. 6, n. 3, p. 667-674, 2007.
WOS000251696600023.pdf
1676-5680
http://repositorio.unifesp.br/handle/11600/43719
WOS:000251696600023
url http://www.funpecrp.com.br/gmr/year2007/vol3-6/pdf/GMR0370.pdf
http://repositorio.unifesp.br/handle/11600/43719
identifier_str_mv Genetics And Molecular Research. Ribeirao Preto: Funpec-editora, v. 6, n. 3, p. 667-674, 2007.
WOS000251696600023.pdf
1676-5680
WOS:000251696600023
dc.language.iso.fl_str_mv eng
language eng
dc.relation.none.fl_str_mv Genetics And Molecular Research
dc.rights.driver.fl_str_mv info:eu-repo/semantics/openAccess
eu_rights_str_mv openAccess
dc.format.none.fl_str_mv 667-674
application/pdf
dc.publisher.none.fl_str_mv Funpec-editora
publisher.none.fl_str_mv Funpec-editora
dc.source.none.fl_str_mv reponame:Repositório Institucional da UNIFESP
instname:Universidade Federal de São Paulo (UNIFESP)
instacron:UNIFESP
instname_str Universidade Federal de São Paulo (UNIFESP)
instacron_str UNIFESP
institution UNIFESP
reponame_str Repositório Institucional da UNIFESP
collection Repositório Institucional da UNIFESP
repository.name.fl_str_mv Repositório Institucional da UNIFESP - Universidade Federal de São Paulo (UNIFESP)
repository.mail.fl_str_mv biblioteca.csp@unifesp.br
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