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)...

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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

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5751197/
https://www.ncbi.nlm.nih.gov/pubmed/29194406
http://dx.doi.org/10.3390/ijms18122594

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