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Nothing is more hopeful or gratifying for the retinal disease community than an emerging or FDA-approved therapy that provides vision to people with advanced retinal disease. Such is the case [...]

Caitlin and Greg Smith eagerly looked forward to the birth of their fourth child, who would complete their busy family of two girls and a boy. Caitlin’s pregnancy was unremarkable, […]

Hope in Focus will host its signature fundraising event of the year – Dinner in the Dark – on Saturday, Nov. 2, 2024, at Foxwoods Resort Casino in Mashantucket, CT. […]

A $100,000 donation from Sofia Sees Hope will support research into restoring vision for patients diagnosed with Leber congenital amaurosis (LCA). The grant will also provide access to genetic [...]

Mikayla Larson, a 30-year-old mother living with a rare inherited retinal disease (IRD) called Leber congenital amaurosis (LCA) wants to reassure children living with the same disease that [...]

Successful clinical drug trials are a cornerstone of U.S. Food and Drug Administration approval, such as with LUXTURNA™, a ground-breaking genetic therapy that helps restore vision in Leber [...]

Transforming laboratory research into real-life therapy for patients is a rare occurrence. But when it does happen, it’s big. Huge, in fact. Ask Dr. Audina M. Berrocal, the pediatric retinal […]

Gene mutations in the rare inherited retinal disease of Leber congenital amaurosis commonly are referred to by their gene name, such as GUCY2D, RPE65 and CEP290. But sometimes, as LCA patients [...]

By Angélica Bretón Morán I am 22 years old and I have LCA. My name is Angélica Bretón Morán, I am from Mexico, I am 22 years old and I […]

This is the seventh in a series following the progress of Creed Pettit, a 9-year-old Florida third-grader, who completed treatment in March with the breakthrough gene-therapy drug called [...]