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    • Long-term outcomes of systemic therapies for Hurler syndrome : an international multicenter comparison 

      Eisengart, Julie B.; Rudser, Kyle D.; Xue, Yong; Orchard, Paul J.; Miller, Weston P.; Lund, Troy C.; Van Der Ploeg, Ans T.; Mercer, Jean; Jones, Simon A.; Mengel, Karl Eugen; Gökce, Seyfullah; Guffon, Nathalie; Giugliani, Roberto; Souza, Carolina Fischinger Moura de; Shapiro, Elsa G.; Whitley, Chester B. (2018) [Journal article]
      Purpose: Early treatment is critical for mucopolysaccharidosis type I (MPS I), justifying its incorporation into newborn screening. Enzyme replacement therapy (ERT) treats MPS I, yet presumptions that ERT cannot penetrate ...