DOI: 10.1055/s-00000140

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References

Russell S, Bennett J, Wellman JA. et al.
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

Lancet 2017;
390: 849-860
doi:10.1016/S0140-6736(17)31868-8

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