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

JB, Eisengart; KD, Rudser; Y, Xue; P, Orchard; W, Miller; T, Lund; der Ploeg A, Van; J, Mercer; S, Jones; KE, Mengel; S, Gökce; N, Guffon; R, Giugliani; Souza CFM, de; EG, Shapiro; CB, Whitley

doi:10.1038/gim.2018.29

Public

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

Shared by NobleBlocks on Mar 8, 2018 • 12:00 AM UTC

Authors:

Eisengart JB

Rudser KD

Xue Y

Abstract

<h4>Purpose</h4>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 the blood-brain barrier (BBB) support recommendations that hematopoieti...

Subject

Mucopolysaccharidosis

Enzyme Replacement Therapy

Neurodegenerative

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