Intravenous AVI4658 Shows Safety, Benefit in DMD

AVI BioPharma reports intravenous AVI4658 appears safe and beneficial to those with DMD who have mutations in or around exon 51, following a 26-week study

• Nineteen people with DMD with mutations in the area of exon 51 of the dystrophin gene received weekly infusions of AVI4658 for 12 weeks and were followed for an additional 14 weeks.
  
• AVI4658 is designed to cause skipping of exon 51 of the dystrophin gene and to cause synthesis of functional dystrophin protein.
  
• The drug was well tolerated, caused substantial dystrophin production in some participants as measured by muscle biopsies at week 14, and did not result in an immune response to the newly made dystrophin.
  
• A phase 2 trial that will test higher doses is being planned.
  

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