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Delivery of therapeutic compounds to the brain and other tissues

  • US 7,442,372 B2
  • Filed: 08/29/2003
  • Issued: 10/28/2008
  • Est. Priority Date: 08/29/2003
  • Status: Active Grant
First Claim
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1. A method for treating a mammal suffering from a lysosomal storage disease of the brain, spinal cord or meninges associated with diminished activity of an enzyme comprising intrathecally administering directly into the cerebrospinal fluid (CSF) of the mammal a pharmaceutical composition comprising said enzyme in an amount effective to ameliorate symptoms of said lysosomal storage disease, wherein said symptoms are selected from the group consisting of an increased amount of storage granules, an increased amount of glycosaminoglycans, meningeal swelling, spinal cord compression and nystagmus.

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