Safety, pharmacokinetics and CNS distribution of tralesinidase alfa administered via intracerebroventricular infusion to juvenile cynomolgus monkeys

Mucopolysaccharidosis Type IIIB (MPS IIIB) is an ultrarare, fatal pediatric disease with no approved therapy. It is caused by mutations in the gene encoding for lysosomal enzyme alpha-N-acetylglucosaminidase (NAGLU). Tralesinidase alfa (TA) is a fusion protein comprised of recombinant NAGLU and a mo...

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Detalles Bibliográficos
Autores principales: Pinkstaff, Jason, McCullagh, Emma, Grover, Anita, Melton, Andrew C., Cherukuri, Anu, Wait, Jill CM, Nguyen, Annalisa, Butt, Mark T., Trombley, Jami L., Reed, Randall P., Adams, Eric.L., Boyd, Robert B., Chandra, Sundeep, Henshaw, Joshua, O’Neill, Charles A., Zanelli, Eric, Kovalchin, Joseph
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Elsevier 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10009680/
https://www.ncbi.nlm.nih.gov/pubmed/36923444
http://dx.doi.org/10.1016/j.toxrep.2023.02.014

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