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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التفاصيل البيبلوغرافية

المؤلفون الرئيسيون:	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
التنسيق:	Artigo
اللغة:	Inglês
منشور في:	Elsevier 2023-01-01
سلاسل:	Toxicology Reports
الموضوعات:	MPS IIIB Sanfilippo syndrome NAGLU Intracerebroventricular Enzyme replacement therapy
الوصول للمادة أونلاين:	http://www.sciencedirect.com/science/article/pii/S2214750023000288
الوسوم:	إضافة وسم لا توجد وسوم, كن أول من يضع وسما على هذه التسجيلة!