Whole Body and CNS Biodistribution of rhHNS in Cynomolgus Monkeys after Intrathecal Lumbar Administration: Treatment Implications for Patients with MPS IIIA

Mucopolysaccharidosis III type A (MPS IIIA; Sanfilippo syndrome), a genetic lysosomal disorder causing a deficiency of heparan N-sulfatase (HNS), leads to progressive cognitive decline from an early age. An effective enzyme replacement therapy (ERT) for MPS IIIA requires central nervous system (CNS)...

Descripción completa

Detalles Bibliográficos
Autores principales: Chung, Jou-Ku, Pan, Luying, Palmieri, Kathleen, Youssef, Amir S., McCauley, Thomas G.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2017
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5751197/
https://www.ncbi.nlm.nih.gov/pubmed/29194406
http://dx.doi.org/10.3390/ijms18122594

Ejemplares similares

Ejemplares similares