Jonathan Heller,Mohammed Qatanani,Gregory Grabowski
申请号：
US15525690
公开号：
US20180185495A1
申请日:
2015.11.10
申请国别(地区):
US
年份:
2018
代理人:
摘要：
The present invention provides pharmaceutical compositions comprising an a blood brain barrier peptide and a human peptide, such as an alpha-L-iduronidase (IDUA) protein, an iduronate-2-sulfatase protein (IDS) protein, or an a galactosidase A protein (α-Gal A) protein. The invention further provides methods of use for treating Mucopolysaccharidosis type I (MPS I), including Hurler Syndrome, Hurler-Scheie Syndrome and Scheie Syndrome methods of use for treating Hunter syndrome and methods of use for treating Fabry disease.
