Concepedia

Publication | Open Access

Enzyme-Replacement Therapy in Mucopolysaccharidosis I

DOIFull Paper Access

670

Citations

19

References

2001

Year

Emil Kakkis, Joseph Muenzer, George E. Tiller, Lewis Waber, John W. Belmont, Merry Passage, Barbara Izykowski, J. Duncan Phillips, Robin Winkler Doroshow, Irv Walot,
New England Journal of Medicine

Abstract

In patients with mucopolysaccharidosis I, treatment with recombinant human alpha-L-iduronidase reduces lysosomal storage in the liver and ameliorates some clinical manifestations of the disease.

References

YearCitations

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