Article
Oral 9-cis retinoid for childhood blindness due to Leber congenital amaurosis caused by RPE65 or LRAT mutations: an open-label phase 1b trial.
Lancet (London, England) - 25 Oct 2014
Koenekoop Robert K, Sui Ruifang, Sallum Juliana, van den Born L Ingeborgh, Ajlan Radwan, Khan Ayesha, den Hollander Anneke I, Cremers Frans P M, Mendola Janine D, Bittner Ava K, Dagnelie Gislin, Schuchard Ronald A, Saperstein David A
Abstract excerpt
BACKGROUND: Leber congenital amaurosis, caused by mutations in RPE65 and LRAT, is a severe form of inherited retinal degeneration leading to blindness. We aimed to assess replacement of the missing chromophore 11-cis retinal with oral QLT091001 (synthetic 9-cis-retinyl acetate) in these patients. METHODS: In our open-label, prospective, phase 1b trial, we enrolled patients (aged ≥6 years) with Leber congenital...
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