Article ID Journal Published Year Pages File Type
8342920 Molecular Genetics and Metabolism 2018 12 Pages PDF
Abstract
ERT improves uGAGs and liver/spleen volume with a moderate evidence level in MPS-II patients initiating therapy as adults, although the putative associated clinical benefit is unclear.
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Life Sciences Biochemistry, Genetics and Molecular Biology Biochemistry
Preview
Efficacy of Idursulfase therapy in patients with Mucopolysaccharidosis type II who initiated enzyme replacement therapy in adult age. A systematic review of the literature
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