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Exon Skipping Therapy for Functional Amelioration of Semifunctional Dystrophin in Becker and Duchenne Muscular Dystrophy

  • US 20120172415A1
  • Filed: 08/31/2010
  • Published: 07/05/2012
  • Est. Priority Date: 08/31/2009
  • Status: Abandoned Application
First Claim
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1. A method for treating a muscular dystrophy caused by instability of a semi-functional dystrophin in a patient in need thereof, comprising the step ofadministering to said patient antisense oligonucleotides complementary to nucleic acid sequences that are necessary for correct splicing of a region comprising or consisting of exon 42 of said semi-functional dystrophin.

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