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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 […]

This is the fifth 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™, [...]

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 […]