Open AccessOutcome Measures for Clinical Trials of Leber Congenital Amaurosis Caused by the Intronic Mutation in the CEP290 Gene
Author(s) -
Samuel G. Jacobson,
Artur V. Cideciyan,
Alexander Sumaroka,
Alejandro J. Román,
Jason Charng,
Monica Lu,
Windy Choi,
Rebecca Sheplock,
Małgorzata Świder,
Mychajlo S. Kosyk,
Sharon Schwartz,
Edwin M. Stone,
Gerald A. Fishman
Publication year - 2017
Publication title -
investigative ophthalmology and visual science
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.935
H-Index - 218
eISSN - 1552-5783
pISSN - 0146-0404
DOI - 10.1167/iovs.17-21560
Subject(s) - ophthalmology , electroretinography , medicine , retinal , retina , biology , neuroscience
To determine efficacy outcome measures for clinical trials of Leber congenital amaurosis (LCA) associated with a common intronic mutation in the CEP290 gene.
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