Retinal Dystrophy Associated With RLBP1 Retinitis Pigmentosa: A Five-Year Prospective Natural History Study
Investigative Ophthalmology & Visual Science2023Vol. 64(13), pp. 42–42
Citations Over TimeTop 22% of 2023 papers
Marie Burstedt, James Whelan, Jane S. Green, Karen Holopigian, Claudio Spera, Erin Greco, Jean-Yves Deslandes, Michael Wald, Cynthia L. Grosskreutz, Xiao Ni, Guillaume Normand, Michael Maker, A. Charil, Michael Rosol, Yunsheng He, Kalliopi Stasi
Abstract
This natural history study, which is the first study to monitor patients with RLBP1 RD for five years, showed that severely delayed DA sensitivity recovery, a characteristic feature of this disease, was observed in all patients across all age groups (17-69 years), making it a potentially suitable efficacy assessment for gene therapy treatment in this patient population.
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