Patent ID: 9089566
Filing Date: 2015-07-28
Classification: A61K,A61P,C12Y

Abstract:
1. A method for treating Sanfilippo syndrome type B (Mucopolysaccharidosis type IIIB or MPS IIIB) comprising the step of administering intrathecally to a human subject in need of treatment a pharmaceutical composition comprising a fusion protein comprising recombinant human α-N-acetylglucosaminidase and a moiety that facilitates high uptake of a lysosomal enzyme, wherein the moiety is an IGF-II polypeptide that allows the lysosomal enzyme to bind to the mannose-6-phosphate receptor, and wherein the fusion protein is administered in an amount effective to ameliorate one or more central nervous system (CNS) symptoms of Sanfilippo syndrome type B.