The present invention provides methods for treating Pompe disease in a subject by administering to the subject a therapeutically effective amount of a fusion protein which includes human acid alpha-glucosidase (GAA), or a fragment thereof, and a lysosomal targeting domain. The lysosomal targeting domain binds the human cation-independent mannose-6-phosphate receptor in a mannose-6-phosphate-independent manner.
申请公布号
WO2008063511(A3)
申请公布日期
2009.04.09
申请号
WO2007US23881
申请日期
2007.11.13
申请人
ZYSTOR THERAPEUTICS, INC.;LEBOWITZ, JONATHAN;MAGA, JOHN