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SPK-RPE65 meets primary endpoint in phase 3 pivotal trial
SPK-RPE65, a potential gene therapy treatment for RPE65-mediated inherited retinal dystrophies, has met its primary endpoint of demonstrating improvement in visual function as measured by the change in bilateral mobility testing between baseline and 1 year in a phase 3 pivotal trial, according to a press release from Spark Therapeutics. Patients who received SPK-RPE65 outperformed controls in the two secondary endpoints of full-light sensitivity threshold testing (P < .001) and mobility test change score for the first injected eye (P = .001). The third secondary endpoint of visual acuity did (Read more...)