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POLARIS (SB-SC-001): An Observational Human Study in Pediatric and Adult Subjects to Follow the Progression of Stargardt Disease (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ATP Binding Cassette Subfamily A Member 4 (ABCA4) Gene (Polaris (SB-SC-001))
This is a prospective, observational, non-interventional, multi-center, global clinical study of subjects with early to advanced stage STGD1 caused by bi-allelic likely pathogenic or pathogenic variants in the ABCA4 gene, confirmed genotypically by an accredited genotyping laboratory. The study will consist of 6 visits over a 96-week study period: Visit 1 (Screening Visit), Visit 2 (Week 16), Visit 3 (Week 32) Visit 4 (Week 48), Visit 5 (Week 72), and Visit 6 (Week 96).
Subjects who discontinue the study early will ...
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