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To evaluate disease progression in a cohort of patients with a clinical and genetic diagnosis of Stargardt disease.Longitudinal cohort study.A total of 56 selected patients with a clinical and molecular diagnosis of Stargardt disease, an early age of onset, and a median follow-up length of 2 years.Patients underwent routine examination, including full-field electroretinography, microperimetry, and...
The aim of this study was to show the clinical data of long-term (3-year) follow-up of 5 patients affected by Leber congenital amaurosis type 2 (LCA2) treated with a single unilateral injection of adeno-associated virus AAV2-hRPE65v2. Clinical trial. Five LCA2 patients with RPE65 gene mutations. After informed consent and confirmation of trial eligibility criteria, the eye with worse visual...
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