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SUBSTRATE REDUCTION THERAPY

  • US 20140080769A1
  • Filed: 09/13/2013
  • Published: 03/20/2014
  • Est. Priority Date: 06/27/2007
  • Status: Active Grant
First Claim
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1. A method of treating a subject having a disease which has a secondary Niemann-Pick type C disease like cellular phenotype, provided that the disease is other than mucopolysaccharidosis and other than mucolipidosis IV, wherein the method comprises selecting the subject having the disease, and administering to the subject an effective amount of an inhibitor of sphingolipid biosynthesis.

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