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

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

Abstract 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 (...

Full description

Bibliographic Details
Main Authors: 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
Format: Article
Language:English
Published: BMC 2018-07-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Mucopolysaccharidosis Type I
Iduronidase
Blood-brain barrier
Insulin receptor
Open label clinical trial
Safety
Online Access:http://link.springer.com/article/10.1186/s13023-018-0849-8

Internet

http://link.springer.com/article/10.1186/s13023-018-0849-8

Similar Items