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

Autor: 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

Publikováno v: Toxicology Reports, Vol 10, Iss , Pp 357-366 (2023)

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 fusi

Externí odkaz: https://doaj.org/article/cfbd12d153c245b5adea0f11f64be6c8

Intravitreal enzyme replacement inhibits progression of retinal degeneration in canine CLN2 neuronal ceroid lipofuscinosis

Autor: Rebecca E.H. Whiting, Annalisa Nguyen, Charles A. O'Neill, Martin L. Katz, Joseph B. Kowal, Cheryl A. Jensen, Stefanie Lim, Carley R. Corado, Grace Robinson Kick, Leilani J. Castaner, Juri Ota-Kuroki

Publikováno v: Exp Eye Res

CLN2 neuronal ceroid lipofuscinosis is a rare recessive hereditary retinal and neurodegenerative disease resulting from deleterious sequence variants in TPP1 that encodes the soluble lysosomal enzyme tripeptidyl peptidase-1 (TPP1). Children with this

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=doi_dedup___::6981cfc8ea142c1b7a7a2b81c2067604
https://pubmed.ncbi.nlm.nih.gov/32634395

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

Autor: Pinkstaff J; BioMarin Pharmaceutical Inc., Novato, CA, USA., McCullagh E; BioMarin Pharmaceutical Inc., Novato, CA, USA., Grover A; BioMarin Pharmaceutical Inc., Novato, CA, USA., Melton AC; BioMarin Pharmaceutical Inc., Novato, CA, USA., Cherukuri A; BioMarin Pharmaceutical Inc., Novato, CA, USA., Wait JC; BioMarin Pharmaceutical Inc., Novato, CA, USA., Nguyen A; BioMarin Pharmaceutical Inc., Novato, CA, USA., Butt MT; StageBio, Mt. Jackson, VA, USA., Trombley JL; Northern Biomedical Research Inc., Norton Shores, MI, USA., Reed RP; Northern Biomedical Research Inc., Norton Shores, MI, USA., Adams EL; Northern Biomedical Research Inc., Norton Shores, MI, USA., Boyd RB; Northern Biomedical Research Inc., Norton Shores, MI, USA., Chandra S; BioMarin Pharmaceutical Inc., Novato, CA, USA., Henshaw J; BioMarin Pharmaceutical Inc., Novato, CA, USA., O'Neill CA; BioMarin Pharmaceutical Inc., Novato, CA, USA., Zanelli E; Allievex Corporation, Marblehead, MA, USA., Kovalchin J; Allievex Corporation, Marblehead, MA, USA.

Publikováno v: Toxicology reports [Toxicol Rep] 2023 Mar 01; Vol. 10, pp. 357-366. Date of Electronic Publication: 2023 Mar 01 (Print Publication: 2023).