| abstract |
Methods and compositions for CNS delivery of iduronate-2-sulfatase. The invention provides, among other things, compositions and methods for CNS delivery of lysosomal enzymes for the effective treatment of lysosomal storage diseases. In some embodiments, the invention provides direct CNS intrathecal administration comprising iduronate-2-sulfatase (I2S) protein, a salt, and a polysorbate surfactant for the treatment of Hunter syndrome. For stable formulations. In various aspects, the invention includes a method of treating Hunter's syndrome comprising the step of intrathecal administration of a formulation according to any of the embodiments described herein to a subject in need thereof. To do. [Selection diagram] None |