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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Dettagli Bibliografici
Pubblicato in:Rev Paul Pediatr
Autori principali: 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
Natura: Artigo
Lingua:Inglês
Pubblicazione: Sociedade de Pediatria de São Paulo 2019
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Original Articles
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC6868559/
https://ncbi.nlm.nih.gov/pubmed/31090850
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1590/1984-0462/;2019;37;3;00010
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