Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study

Agnes H. Chen, Paul Harmatz, Igor Nestrasil, Julie B. Eisengart, Kelly E. King, Kyle Rudser, Alexander M. Kaizer, Alena Svatkova, Amy Wakumoto, Steven Q. Le, Jacqueline Madden, Sarah Young, Haoyue Zhang, Lynda E. Polgreen, Patricia I. Dickson

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