Visual acuity rates and full-field light sensitivity improved after treatment with intravitreal sepofarsen in patients with Leber congenital amaurosis 10 due to the p.Cys998X mutation in the CEP290 gene.
“The most encouraging aspect of this trial was the surprising finding of mean improvements of visual acuity and full-field light sensitivity and, in some instances, unexpectedly outsized improvements even in subjects with baseline light perception vision,” Stephen R. Russell, MD, said at the virtual Association for Research in Vision and Ophthalmology meeting.
The 12-month
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