Intrathecal enzyme replacement for cognitive decline in mucopolysaccharidosis type I, a randomized, open-label, controlled pilot study.
Molecular Genetics And Metabolism
Chen, Agnes H AH; Harmatz, Paul P; Nestrasil, Igor I; Eisengart, Julie B JB; King, Kelly E KE; Rudser, Kyle K; Kaizer, Alexander M AM; Svatkova, Alena A; Wakumoto, Amy A; Le, Steven Q SQ; Madden, Jacqueline J; Young, Sarah S; Zhang, Haoyue H; Polgreen, Lynda E LE; Dickson, Patricia I PI
Genotype-phenotype relationships in mucopolysaccharidosis type I (MPS I): Insights from the International MPS I Registry.
Clinical Genetics
Clarke, Lorne A LA; Giugliani, Roberto R; Guffon, Nathalie N; Jones, Simon A SA; Keenan, Hillary A HA; Munoz-Rojas, Maria V MV; Okuyama, Torayuki T; Viskochil, David D; Whitley, Chester B CB; Wijburg, Frits A FA; Muenzer, Joseph J