Arlene Drack, MD, presented data from the Phase III gene replacement trial for RPE65 associated Leber Congenital Amaurosis at the annual meeting of the American Association for Pediatric Ophthalmology and Strabismus held in Vancouver, BC, Canada on April 9, 2016. Results of the trial, carried out at the University of Iowa and the Children's Hospital of Philadelphia (sponsored by Spark Therapeutics), showed statistically significant improvement in navigating a mobility course in treated patients. Many patients could navigate even in very dim light after the treatment. Ability of the retina to detect light was also significantly improved. Visual acuity improved in some, but not all patients. This paper presentation was voted #2 most important overall for the annual meeting.
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