RPE65 and retinitis pigmentosa 1: In summary, the results of this open-label proof-of-concept study using one 7-day oral dose of QLT091001 demonstrate an acceptable safety profile and an improvement in the visual field and/or visual acuity in a majority of patients with RP due to mutations in RPE65 and LRAT. A subset of patients who underwent fMRI showed an increase in visual cortical activation after treatment.