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CASE SERIES OF PATIENTS UNDER BIWEEKLY TREATMENT WITH LARONIDASE: A REPORT OF A SINGLE CENTER EXPERIENCE

OBJECTIVE: To report the stabilization of urinary glycosaminoglicans (GAG) excretion and clinical improvements in patients with mucopolysaccharidosis type I (MPS I) under an alternative dose regimen of laronidase of 1.2 mg/kg every other week. METHODS: We participated in a dose-optimization trial fo...

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Detalles Bibliográficos
Autores principales:	Kyosen, Sandra Obikawa, Toma, Leny, Nader, Helena Bonciani, Braga, Marion Coting, Pereira, Vanessa Gonçalves, Canossa, Sueli, Pesquero, João Bosco, D’Almeida, Vânia, Martins, Ana Maria
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Sociedade de Pediatria de São Paulo 2019
Materias:	Original Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6868559/ https://www.ncbi.nlm.nih.gov/pubmed/31090850 http://dx.doi.org/10.1590/1984-0462/;2019;37;3;00010

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