Study ID: MTVS-201
Study Title: A Study to Validate the Multi-Luminance Mobility Test (MLMT) for Assessing Functional Vision in Subjects with Inherited Retinal Dystrophies.

This report details the prospective, observational study conducted to validate the MLMT as a primary efficacy endpoint. The study enrolled 60 subjects, including 29 with normal vision and 31 with visual impairment. The MLMT demonstrated the ability to distinguish between normal and visually impaired subjects, showed a high degree of inter-rater reliability (97.9%), and correlated with other standard vision assessments like visual acuity. The study concluded that the MLMT is a reliable, valid, and clinically meaningful measure of functional vision suitable for use as a primary endpoint in clinical trials for retinal dystrophies.

Study ID: RPE65-NHS-001
Study Title: A Retrospective Natural History Study of Patients with Retinal Degeneration Due to Autosomal Recessive Mutations in the RPE65 Gene.

This report describes a retrospective chart review of 70 subjects with RPE65-mediated retinal dystrophy. The study documented the progressive nature of the disease, showing a non-linear decline in visual acuity and visual field with age, leading to total blindness in early adulthood. This study formally characterized the severe and progressive nature of the disease and established the significant unmet medical need for an effective therapy.