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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 [...]

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 [...]

In the sometimes-isolating world of rare inherited retinal diseases (IRDs) such as Leber congenital amaurosis (LCA), another option exists to help bring people together – support by telephone. [...]

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™, [...]

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 [...]

Christian Guardino underwent experimental eye surgery five years ago with a revolutionary genetic treatment called LUXTURNA™, forever changing his life by restoring his vision that was lost due [...]