The results of a phase 2 multicenter trial showed dose-dependent RPE65 inhibition efficacy of emixustat in patients with macular atrophy secondary to Stargardt disease. The 10 mg dose showed near-complete suppression of the rod b-wave recovery rate after photobleaching and was therefore selected for an ongoing phase 3 trial.
“Stargardt disease is the most common form of juvenile macular degeneration, characterized by deposition of toxic bisretinoids at the level of RPE. It is a rare disease for which no approved therapies are available,” Jeff Gregory, MD, said in a presentation (Read more...)
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