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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 [...]

A revolutionary genetic treatment could improve the vision of 5-year-old Maverick Johnston, but his mom first wants to know more about the side effects and capabilities of the breakthrough drug [...]

This is the sixth 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 LUXTURNA™, [...]

Now that LUXTURNA™ has come to market as a revolutionary vision-restoring genetic treatment, how does it get to patients? The answer to that question and many others can be found within […]