Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: An open-label phase 1b trial

Robert K. Koenekoop, Ruifang Sui, Juliana Sallum, L. Ingeborgh Van Den Born, Radwan Ajlan, Ayesha Khan, Anneke I. Den Hollander, Frans P.M. Cremers, Janine D. Mendola, Ava K. Bittner, Gislin Dagnelie, Ronald A. Schuchard, David A. Saperstein

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