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Molecular Therapy. Methods & Clinical Development|July 1, 2017
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B MiceMika Aoyagi-Scharber, Danielle Crippen-Harmon, Roger Lawrence, et al.Drug Delivery and Translational Research|January 17, 2020
Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type BAnita Grover, Danielle Crippen-Harmon, Lacey Nave, et al.The Journal of Biological Chemistry|October 28, 2022
Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunctionJenna Magat, Samantha Jones, Brian Baridon, et al.Proceedings of the National Academy of Sciences of the United States of America|October 1, 2014
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIBShih-Hsin Kan, Mika Aoyagi-Scharber, Steven Q Le, et al.The Journal of Pharmacology and Experimental Therapeutics|June 18, 2022
Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIBN Matthew Ellinwood, Bethann N Valentine, Andrew S Hess, et al.Pageof 4