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COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT

  • US 20160002627A1
  • Filed: 01/09/2014
  • Published: 01/07/2016
  • Est. Priority Date: 01/09/2013
  • Status: Active Grant
First Claim
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1. A method of treating a human patient having spinal muscular atrophy comprising administering to the human patient at least one dose of an antisense compound comprising an antisense oligonucleotide complementary to intron 7 of a nucleic acid encoding human SMN2 pre-mRNA, wherein the antisense compound is administered into the cerebrospinal fluid at a dose of 0.1 to 20 milligrams;

  • and thereby ameliorating at least one symptom of spinal muscular atrophy in the human patient.

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