Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type i after 52 weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): An open label phase 1-2 trial
Roberto Giugliani, Luciana Giugliani, Fabiano De Oliveira Poswar, Karina Carvalho Donis, Amauri Dalla Corte, Mathias Schmidt, Ruben J. Boado, Igor Nestrasil, Carol Nguyen, Steven Chen, William M. Pardridge
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