Skipping Exon 51 • Drisapersen – proteinuria and skin scars in injection site • Pts deteriorated following trial discontinuation
• Sarepta (previously AVI)- Eteplirsen (morpholino oligonucleotides) met the primary efficacy endpoints: increase in novel dystrophin (47% of normal) and significant clinical benefit in 6MWT (-42.3 to +16.5 m) in children less than 9.5 years. • Plans for 45, 50, 53