scholarly journals Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

The Lancet ◽  
2017 ◽  
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Jennifer A Wellman ◽  
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Atsuo Taniguchi ◽  
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Vol 54 (1) ◽  
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