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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