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Efficacy and safety of intravenous laronidase for mucopolysaccharidosis type I: A systematic review and meta-analysis.

Alícia Dorneles DornellesOsvaldo ArtigalásAndré Anjos da SilvaDora Lucia Vallejo ArdilaTaciane AlegraTiago Veiga PereiraFilippo Pinto E VairoIda Vanessa Doederlein Schwartz
Published in: PloS one (2017)
Our findings suggest that IV laronidase effectively reduces urinary GAGs excretion, hepatomegaly and LVMI, and can improve shoulder flexion in MPS I patients. Laronidase appears to be safe in the studied population.
Keyphrases
  • end stage renal disease
  • ejection fraction
  • newly diagnosed
  • chronic kidney disease
  • peritoneal dialysis
  • prognostic factors
  • high dose
  • replacement therapy
  • rotator cuff