| 摘要 |
<p>The present invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for effective treatment of lysosomal storage diseases. In some embodiments, the present invention includes a stable formulation for direct CNS intrathecal administration comprising a heparan N-sulfatase (HNS) protein, salt, and a polysorbate surfactant for the treatment of Sanfilippo Syndrome Type A.</p> |
| 申请人 |
SHIRE HUMAN GENETIC THERAPIES, INC. |
发明人 |
NATOLI, FARAH;ZHU, GAOZHONG;TEREW, JENNIFER;JIANG, YUAN;TSUNG, JAMIE;SHAHROKH, ZAHRA;VERNAGLIA, BRIAN;PAN, JING;PFEIFER, RICHARD;CALIAS, PERICLES |
