Laronidase for treating mucopolysaccharidosis type I
Autor(a) principal: | |
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Data de Publicação: | 2007 |
Outros Autores: | |
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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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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1814268377764462592 |