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Gene therapy shows gain in functional vision in continuation of phase 3 trial
Voretigene neparvovec demonstrated a gain in functional vision in patients with inherited retinal disease caused by the RPE65 gene, Spark Therapeutics announced in a press release.The phase 3 trial included 20 patients who received voretigene neparvovec, formerly SPK-RPE65, in both eyes (intervention group) and a control group of nine patients. The primary endpoint of the randomized, controlled portion of the trial was change in bilateral mobility testing from baseline to 1 year. Spark previously announced a statistically and clinically significant improvement in bilateral mobility testing in treated patients compared with (Read more...)