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See Latest ArticlesLocal restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study
The Lancet Neurology, 09/10/09
Kinali M et al. – Intramuscular AVI–4658 was safe and induced the expression of dystrophin locally within treated muscles. This proof–of–concept study has led to an ongoing systemic clinical trial of AVI–4658 in patients with DMD.
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