METHODS AND COMPOSITIONS FOR THE TREATMENT OF TYPE IIIA MUCOPOLISOHARIDOSIS USING INTRATECAL INTRODUCTION

摘要

The present invention, among other things, proposes an effective method of treating Sanfillipo-type A syndrome (MPS IIIA), based on the intrathecal delivery of the recombinant heparin-N-sulfatase enzyme (HNS). In some embodiments of the invention, the present invention includes methods for treating Sanfillipo type A syndrome (MPS IIIA) by intrathecal administration of a recombinant HNS enzyme in a therapeutically effective dose and with a therapeutically effective administration interval for a period sufficient to reduce the level of glycosaminoglycan (GAG) heparan sulfate - cerebral fluid (CSF) and / or urine compared to control.