Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes

Eisengart, Julie B.; Pierpont, Elizabeth I.; Kaizer, Alexander M.; Rudser, Kyle D.; King, Kelly E.; Pasquali, Marzia; Polgreen, Lynda E.; Dickson, Patricia I.; Le, Steven Q.; Miller, Weston P.; Tolar, Jakub; Orchard, Paul J.; Lund, Troy C.

期刊名称Genetics in Medicine
作者Eisengart, Julie B.; Pierpont, Elizabeth I.; Kaizer, Alexander M.; Rudser, Kyle D.; King, Kelly E.; Pasquali, Marzia; Polgreen, Lynda E.; Dickson, Patricia I.; Le, Steven Q.; Miller, Weston P.; Tolar, Jakub; Orchard, Paul J.; Lund, Troy C.
印刷ISSN1098-3600
电子ISSN1530-0366
文件大小778.56KB
发表时间2019-4-25
DOI10.1038/s41436-019-0522-1

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Intrathecal enzyme replacement for Hurler syndrome: biomarker association with neurocognitive outcomes