| 摘要 |
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 an arylsulfatase A (ASA) protein, salt, and a polysorbate surfactant for the treatment of Metachromatic Leukodystrophy Disease. |
| 申请人 |
SHIRE HUMAN GENETIC THERAPIES, INC.;SALAMAT-MILLER, NAZILA;TAYLOR, KATHERINE;CAMPOLIETO, PAUL;SHAHROKH, ZAHRA;PAN, JING;CHARNAS, LAWRENCE;WRIGHT, TERESA LEAH;CALIAS, PERICLES |
发明人 |
SALAMAT-MILLER, NAZILA;TAYLOR, KATHERINE;CAMPOLIETO, PAUL;SHAHROKH, ZAHRA;PAN, JING;CHARNAS, LAWRENCE;WRIGHT, TERESA LEAH;CALIAS, PERICLES |
