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

• Egile Nagusiak: Sandra Obikawa Kyosen (Egilea), Leny Toma (Egilea), Helena Bonciani Nader (Egilea), Marion Coting Braga (Egilea), Vanessa Gonçalves Pereira (Egilea), Sueli Canossa (Egilea), João Bosco Pesquero (Egilea), Vânia D'Almeida (Egilea), Ana Maria Martins (Egilea)
• Formatua: Liburua
• Argitaratua: Sociedade de Pediatria de São Paulo, 2019-05-01T00:00:00Z.
• Gaiak: article
• Sarrera elektronikoa: Connect to this object online.

ABSTRACT 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...