Evaluation of non-reducing end pathologic glycosaminoglycan detection method for monitoring therapeutic response to enzyme replacement therapy in human mucopolysaccharidosis I.

Vera, Moin U; Le, Steven Q; Victoroff, Alla; Passage, Merry B; Brown, Jillian R; Crawford, Brett E; Polgreen, Lynda E; Chen, Agnes H; Dickson, Patricia I

Vera, Moin U; Le, Steven Q; Victoroff, Alla; Passage, Merry B; Brown, Jillian R; Crawford, Brett E; Polgreen, Lynda E; Chen, Agnes H; Dickson, Patricia I.

Afiliação

Vera MU; Children's Hospital Los Angeles, Los Angeles, CA, USA; Department of Pediatrics, Los Angeles Biomedical Research Institute, Harbor-UCLA Medical Center, Torrance, CA, USA.
Le SQ; Department of Pediatrics, Los Angeles Biomedical Research Institute, Harbor-UCLA Medical Center, Torrance, CA, USA; Department of Pediatrics, Washington University in St. Louis, St. Louis, MO, USA.
Victoroff A; Children's Hospital Los Angeles, Los Angeles, CA, USA.
Passage MB; Department of Pediatrics, Los Angeles Biomedical Research Institute, Harbor-UCLA Medical Center, Torrance, CA, USA.
Brown JR; TEGA Therapeutics, Inc., San Diego, CA, USA.
Crawford BE; BioMarin Pharmaceutical Inc., San Rafael, CA, USA.
Polgreen LE; Department of Pediatrics, Los Angeles Biomedical Research Institute, Harbor-UCLA Medical Center, Torrance, CA, USA.
Chen AH; Department of Pediatrics, Los Angeles Biomedical Research Institute, Harbor-UCLA Medical Center, Torrance, CA, USA.
Dickson PI; Department of Pediatrics, Los Angeles Biomedical Research Institute, Harbor-UCLA Medical Center, Torrance, CA, USA; Department of Pediatrics, Washington University in St. Louis, St. Louis, MO, USA. Electronic address: pdickson@wustl.edu.

Mol Genet Metab ; 129(2): 91-97, 2020 02.

Article em En | MEDLINE | ID: mdl-31630958

Assuntos

Terapia de Reposição de Enzimas; Glicosaminoglicanos/sangue; Glicosaminoglicanos/urina; Mucopolissacaridose I/tratamento farmacológico; Biomarcadores/sangue; Técnicas de Laboratório Clínico; Monitoramento de Medicamentos/métodos; Glicosaminoglicanos/líquido cefalorraquidiano; Humanos; Iduronidase/genética; Iduronidase/uso terapêutico

Palavras-chave

Enzyme replacement therapy; Glycosaminoglycan; Hurler; Lysosomal storage disorder; Mucopolysaccharidosis

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Texto completo: 1 Base de dados: MEDLINE Assunto principal: Mucopolissacaridose I / Terapia de Reposição de Enzimas / Glicosaminoglicanos Tipo de estudo: Diagnostic_studies / Evaluation_studies Limite: Humans Idioma: En Ano de publicação: 2020 Tipo de documento: Article

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