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

Sandra Obikawa KyosenLeny TomaHelena Bonciani NaderMarion Coting BragaVanessa Gonçalves PereiraSueli CanossaJoão Bosco PesqueroVânia D'AlmeidaAna Maria Martins
Published in: Revista paulista de pediatria : orgao oficial da Sociedade de Pediatria de Sao Paulo (2019)
Patients remained clinically stable under the alternative regimen, did not present elevation of urinary GAG nor any adverse event.Conclusions: The switch of dose regimen to 1.2 mg/kg EOW of laronidase was safe, and did not cause any clinical worsening in patients who had been previously under standard dose ERT.
Keyphrases
  • end stage renal disease
  • chronic kidney disease
  • ejection fraction
  • newly diagnosed
  • peritoneal dialysis
  • prognostic factors
  • emergency department
  • patient reported