Tomas S Aleman, Alejandro J Roman, Katherine E Uyhazi, Yu You Jiang, Emma C Bedoukian, Alexander Sumaroka, Vivian Wu, Malgorzata Swider, Iryna Viarbitskaya, Robert C Russell, Elizabeth O Shagena, Arlene J Santos, Leona W Serrano, Kelsey M Parchinski, Rebecca J Kim, Mariejel L Weber, Alexandra V Garafalo, Dorothy A Thompson, Albert M Maguire, Jean Bennett, Drew H Scoles, Erin C O'Neil, Jessica I W Morgan, Artur V Cideciyan
چکیده
The purpose of this study was to determine the natural history of the photoreceptor disease in a large group of pediatric patients with RHD12-associated Leber congenital amaurosis (RDH12-LCA), to estimate the changes expected over the duration of a clinical trial, and to define the relationship…
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DOI: 10.1167/iovs.65.14.30