FIELD: medicine.
SUBSTANCE: direct delivery to the CNS is performed by intrathecal administration of a composition containing a substitution enzyme to replace the lysosomal enzyme at a concentration above approx. 5 mg/ml and up to 50 mM of phosphate to a subject suffering from or predisposed to a lysosomal storage disease associated with a decrease in lysosomal enzyme level or activity. Methods for intrathecal enzyme delivery, methods for treatment of Hunter syndrome, metachromatic leukodystrophy, type A and type B Sanfilippo syndrome, globoid cell leukodystrophy are also provided.
EFFECT: group of inventions allows direct delivery of compositions with high concentrations of displacing lysosomal enzyme and low concentration of phosphate to the CNS without causing significant side effects.
73 cl, 49 tbl, 192 dwg, 29 ex
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