PHARMACEUTICAL FORMULATION COMPRISING A REPLACEMENT ENZYME FOR A LYSOSOMAL ENZYME FOR USE IN TREATING LYSOSOMAL STORAGE DISEASE INTRATHECALLY

摘要

The present invention provides an effective and less invasive approach for direct delivery of therapeutic agents to the central nervous system (CNS). In some embodiments, the present invention provides methods including a step of administering intrathecally to a subject suffering from or susceptible to a lysosomal storage disease associated with reduced level or activity of a lysosomal enzyme, a composition comprising a replacement enzyme for the lysosomal enzyme.