ProQR clinical trial for Leber’s Congenital Amourosis (LCA) 10
Sepofarsen is designed to repair the CEP290 RNA defect and produce a normal functioning CEP290 protein, which will result in improved vision in participants.
This study is ongoing in a number of clinical centres in North America and Europe, involving 30 patients over a 25 month period.
More information about the Illuminate trial can be found on the ProQR website.