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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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Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Elsevier 2023-01-01
Edice:Toxicology Reports
Témata:
MPS IIIB
Sanfilippo syndrome
NAGLU
Intracerebroventricular
Enzyme replacement therapy
On-line přístup:http://www.sciencedirect.com/science/article/pii/S2214750023000288
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