Article
Safety and Proof-of-Concept Study of Oral QLT091001 in Retinitis Pigmentosa Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT).
PloS one - 1 Jan 2015
Scholl Hendrik P N, Moore Anthony T, Koenekoop Robert K, Wen Yuquan, Fishman Gerald A, van den Born L Ingeborgh, Bittner Ava, Bowles Kristen, Fletcher Emily C, Collison Frederick T, Dagnelie Gislin, Degli Eposti Simona, Michaelides Michel, Saperstein David A, Schuchard Ronald A, Barnes Claire, Zein Wadih, Zobor Ditta, Birch David G, Mendola Janine D, Zrenner Eberhart
Abstract excerpt
UNLABELLED: Restoring vision in inherited retinal degenerations remains an unmet medical need. In mice exhibiting a genetically engineered block of the visual cycle, vision was recently successfully restored by oral administration of 9-cis-retinyl acetate (QLT091001). Safety and visual outcomes of a once-daily oral dose of 40 mg/m2/day QLT091001 for 7 consecutive days was investigated in an international,...
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