| 发明名称 |
INTRAVENTRICULAR ENZYME DELIVERY FOR LYSOSOMAL STORAGE DISEASES |
| 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. |
| 申请公布号 |
US2015313970(A1) |
申请公布日期 |
2015.11.05 |
| 申请号 |
US201414559851 |
申请日期 |
2014.12.03 |
| 申请人 |
Genzyme Corporation |
发明人 |
DODGE James;PASSINI Marco A.;SHIHABUDDIN Lamya;CHENG Seng H. |
| 分类号 |
A61K38/46;A61K38/47;A61K9/00 |
主分类号 |
A61K38/46 |
| 代理机构 |
|
代理人 |
|
| 主权项 |
1. A method of treating a patient with Niemann-Pick A or B disease, comprising:
administering an acid sphingomyelinase to the patient via intraventricular delivery to the brain in an amount sufficient to reduce sphingomyelin levels in said brain. |
| 地址 |
Cambridge MA US |
