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Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study.
Chen AH, Harmatz P, Nestrasil I, Eisengart JB, King KE, Rudser K, Kaizer AM, Svatkova A, Wakumoto A, Le SQ, Madden J, Young S, Zhang H, Polgreen LE, Dickson PI. Chen AH, et al. Among authors: eisengart jb. Mol Genet Metab. 2020 Feb;129(2):80-90. doi: 10.1016/j.ymgme.2019.11.007. Epub 2019 Nov 30. Mol Genet Metab. 2020. PMID: 31839529 Free PMC article. Clinical Trial.
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