Navegação Ciências Biológicas por Assunto "Barreira hematoencefálica"
Resultados 1-2 de 2
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Gene therapy for neuronopathic mucopolysaccharidoses : state of the art
(2021) [Artigo de periódico]The need for long-lasting and transformative therapies for mucopolysaccharidoses (MPS) cannot be understated. Currently, many forms of MPS lack a specific treatment and in other cases available therapies, such as enzyme ... -
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
(2018) [Artigo de periódico]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 ...