| abstract |
Methods and compositions for CNS delivery of arylsulfatase A are provided. The present invention provides compositions and methods for CNS delivery of lysosomal enzymes, particularly for the purpose of effectively treating lysosomal storage diseases. In some embodiments, the invention provides a stable formulation for direct intrathecal CNS administration, comprising an arylsulfatase A (ASA) protein and a salt for the treatment of metachromatic leukodystrophy. , A stable formulation comprising a polysorbate surfactant. In various embodiments, the invention includes a stable formulation for direct intrathecal CNS administration, which comprises a arylsulfatase A (ASA) protein, a salt, and a polysorbate surfactant. . [Selection diagram] None |