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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Principais autores:	Jason Pinkstaff, Emma McCullagh, Anita Grover, Andrew C. Melton, Anu Cherukuri, Jill CM Wait, Annalisa Nguyen, Mark T. Butt, Jami L. Trombley, Randall P. Reed, Eric.L. Adams, Robert B. Boyd, Sundeep Chandra, Joshua Henshaw, Charles A. O’Neill, Eric Zanelli, Joseph Kovalchin
פורמט:	Artigo
שפה:	Inglês
יצא לאור:	Elsevier 2023-01-01
סדרה:	Toxicology Reports
נושאים:	MPS IIIB Sanfilippo syndrome NAGLU Intracerebroventricular Enzyme replacement therapy
גישה מקוונת:	http://www.sciencedirect.com/science/article/pii/S2214750023000288
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