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

Stephen R. Russell, Jean Bennett, Jennifer Wellman, Daniel C. Chung, Zi-Fan Yu, Amy Tillman, Janet Wittes, Julie Pappas, Okan U. Elci, Sarah McCague,

The Lancet · 2017 · 1.9K citations · 36 references

Concepts

References

36