ARVO (2021) Russell SR et al., Safety and efficacy of sepofarsen in the second treated eye in a Ph1b/2 extension trial in Leber Congenital Amaurosis type 10 (LCA10)
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Author(s)
Stephen R. Russell1, Arlene V. Drack1, Artur V. Cideciyan2, Samuel G. Jacobson2, Bart P. Leroy3,4, Wilhelmina den Hollander5, Adriana Hollestein-Havelaar5, Michael R. Schwartz5, Aniz Girach5
1Department of Ophthalmology and Visual Sciences, Carver College of Medicine, University of Iowa, Iowa City, IA, USA; 2Scheie Eye Institute, Department of Ophthalmology, Perelman School of Medicine, University of Pennsylvania, Philadelphia, PA, USA; 3Department of Ophthalmology & Center for Medical Genetics, Ghent University Hospital & Ghent University, Ghent, Belgium; 4Ophthalmic Genetics & Visual Electrophysiology, Division of Ophthalmology, The Children’s Hospital of Philadelphia, PA, USA; 5ProQR Therapeutics, Leiden, the Netherlands
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