Microperimetry is a valuable tool for assessing changes in visual function following voretigene neparvovec-rzyl gene therapy

INTRODUCTION: Gene therapy with voretigene neparvovec-rzyl was approved primarily based on improvements observed in the Multi-Luminance Mobility Test (MLMT), while the full-field stimulus threshold (FST) test, a secondary endpoint, also demonstrated efficacy. However, the MLMT is difficult to apply in routine practice, and the FST does not provide spatial information regarding retinal function, which limits direct structural-function correlation. PURPOSE: Therefore, this study investigated whether microperimetry could serve as a simpler, more practical alternative by evaluating its changes after this gene therapy. METHODS: Mean sensitivity and fixation parameters were evaluated in a cohort of 5 patients (10 eyes) who underwent microperi-metry following gene therapy in São Paulo, Brazil. Results from individual eyes showed consistent improvements in retinal sensitivity and fixation stability. These findings were consistent with improvements in daily functioning-particularly in low-light conditions-reported by the patients during medical history assessment during the routine visits. CONCLUSION: Overall, the findings suggest that microperimetry could be an useful tool for monitoring functional outcomes after gene therapy.