| abstract |
Methods and compositions for CNS delivery of heparan N-sulfatase are provided. The present invention provides compositions and methods for CNS delivery of lysosomal enzymes, particularly for effective treatment of lysosomal storage diseases. In some embodiments, the present invention provides a stable for direct CNS intrathecal administration comprising heparan N-sulfatase (HNS) protein, salt, and polysorbate surfactant for treating San Filippo syndrome type A Preparations. [Selection figure] None |