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In the wake of the December 2017 approval by the U.S. Food and Drug Administration of voretigene neparvovec (LUXTURNA™) to treat vision loss in patients with biallelic RPE65 mutation associated [...]

This is the fourth 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 [...]

Told at age 12 she would be blind by 18 because of her Leber congenital amaurosis, Misty Lovelace of Kentucky participated in the gene therapy trials for LUXTURNA, to treat […]

This is the second 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 [...]

It took more than seven years to get a genetic diagnosis for our daughter. During that time, doctors were pretty sure she had LCA, although we also heard that maybe […]

  Amy Reif still can’t hold back tears when she recounts first hearing that a ground-breaking genetic-therapy treatment targeting her daughter’s LCA-RPE65 gene officially received approval. [...]

This is the first in a series following the progress of Creed Pettit, a 9-year-old Florida third-grader, who is a candidate for the breakthrough gene-therapy drug called LUXTURNA™, approved as [...]

Ledyard, CT (February 6, 2018) — Sofia Sees Hope, an organization dedicated to finding treatments and cures for blindness caused by inherited retinal diseases (IRDs), is helping to ensure that [...]

Sofia Sees Hope launches International Rare Disease Day February campaign — ‘Get Tested, Get Connected’ — to encourage those diagnosed with rare inherited retinal diseases to receive genetic [...]

Applied Genetic Technologies Corporation (AGTC), a clinical stage biotech company that focuses on rare inherited retinal diseases (IRDs), develops therapies that replace “broken” genes with [...]