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Laronidase for treating mucopolysaccharidosis type I
(Funpec-editora, 2007-01-01)
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, ...
Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
(BMC [Commercial Publisher], Orphanet [Associate Organisation], 2020)
Ethical issues related to the access to orphan drugs in Brazil: the case of mucopolysaccharidosis type I
(B M J Publishing GroupLondonInglaterra, 2011)
Alternative laronidase dose regimen for patients with mucopolysaccharidosis I: a multinational, retrospective, chart review case series
(Biomed Central Ltd, 2016)
Background: Enzyme replacement therapy (ERT) with laronidase (recombinant human alpha-L-iduronidase, Aldurazyme (R)) is indicated for non-neurological signs and symptoms of mucopolysaccharidosis type I (MPS I). The approved ...
Terapia de reposição da enzima alfa-L-iduronidase recombinante em pacientes portadores de mucopolissacaridose do tipo I: análise de glicosaminoglicanos urinários e correlações clínicas
(Universidade Federal de São Paulo (UNIFESP), 2009)
Mucopolysaccharidosis type I (MPS I) is a lysosomal storage disease caused by deficiency of -L-iduronidase, which cleaves terminal iduronic acid residues of glycosaminoglycans (GAGs), heparan sulphate (HS) and dermatan ...
Enzyme replacement therapy for Mucopolysaccharidosis Type I among patients followed within the MPS Brazil Network
(Sociedade Brasileira de Genética, 2014)
Enzyme replacement therapy for Mucopolysaccharidosis Type I among patients followed within the MPS Brazil Network
(Sociedade Brasileira de Genética, 2014)