Browsing Health Sciences by Author "Nguyen, Carol"
Now showing items 1-1 of 1
-
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
Giugliani, Roberto; Giugliani, Luciana; Poswar, Fabiano de Oliveira; Donis, Karina Carvalho; Corte, Amauri Dalla; Schmidt, Mathias; Boado, Rubén J.; Nestrašil, Igor; Nguyen, Carol; Chen, Steven D.; Pardridge, William M. (2018) [Journal article]Background: Mucopolysaccharidosis (MPS) Type I (MPSI) is caused by mutations in the gene encoding the lysosomal enzyme, α-L-iduronidase (IDUA), and a majority of patients present with severe neurodegeneration and cognitive ...