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Leber Congenital Amaurosis 10
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Leber Congenital Amaurosis 10 trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Leber Congenital Amaurosis 10 trials you may qualify forThe purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjec…
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, i…
Subjects completing participation in study PQ-110-001 (EudraCT 2017-000813-22 / NCT03140969) will be given the opportunity to enroll into the extension study fo…
The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in…
A prospective natural history study with systematic assessments and uniform follow-up to provide a high-quality dataset for assisting in the design of future cl…