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

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 impairment. Recombinant IDUA does not cross the blood-brain barrier (BBB). To...

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Detalles Bibliográficos
Autores principales: Giugliani, Roberto, Giugliani, Luciana, de Oliveira Poswar, Fabiano, Donis, Karina Carvalho, Corte, Amauri Dalla, Schmidt, Mathias, Boado, Ruben J., Nestrasil, Igor, Nguyen, Carol, Chen, Steven, Pardridge, William M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: BioMed Central 2018
Materias:
Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6034233/
https://www.ncbi.nlm.nih.gov/pubmed/29976218
http://dx.doi.org/10.1186/s13023-018-0849-8

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