Table 1

Clinical characteristics of the eyes at baseline

GenderBCVA (decimal)FST blue
(0 dB=0.01 cd/m2)
FST red (0 dB=0.01 cd/m2)
P1 REFemale0.125−0.83 dB1.5 dB
P1 LEFemale0.16−9.71 dB−1.93 dB
P2 LEFemale0.0250.22 dB3.7 dB
P3 LEFemale0.06−0.72 dB2.73 dB
P4 REFemaleFC−4.15 dB0.85 dB
P4 LEFemaleFC−0.91 dB3.18 dB
P8 LEMale0.2−7.6 dB−4.21 dB
  • Seven eyes of five patients with retinal dystrophy caused by bi-allelic RPE65 mutations have been treated with voretigene neparvovec. In all patients, there was no measurable rod function before the treatment and reduced visual acuity.

  • BCVA, best corrected visual acuity; FC, finger counting; FST, full-field stimulus threshold; LE, left eye; RE, right eye.