James Dodge,Marco A. Passini,Lamya Shihabuddin,Seng H. Cheng
申请号：
US14559851
公开号：
US10080783B2
申请日:
2014.12.03
申请国别(地区):
US
年份:
2018
代理人:
摘要：
Lysosomal storage diseases can be successfully treated using intraventricular delivery of the enzyme which is etiologically deficient in the disease. The administration can be performed slowly to achieve maximum effect. Surprisingly, effects are seen on both sides of the blood-brain barrier, making this an ideal delivery means for lysosomal storage diseases which affect both brain and visceral organs.
