A team led by pediatric ophthalmologist Robert Koenekoop gave the medication to 14 patients with Leber congenital amaurosis (LCA), Discover magazine reports in its current issue. LCA is an inherited disease that causes retinal degeneration leading to blindness.
“This is the first time that an oral drug has improved the visual function of blind patients with LCA,” Koenekoop asserted in a McGill University press release last summer. Koenekoop is director of the McGill Ocular Genetics Laboratory at The Montreal Children’s Hospital and a Professor of Human Genetics, Paediatric Surgery and Ophthalmology at McGill. “It is giving hope to many patients who suffer from this devastating retinal degeneration,” he said.
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