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Leber Congenital Amaurosis (LCA)
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Leber Congenital Amaurosis (LCA) trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Leber Congenital Amaurosis (LCA) 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…
The purpose of the study is to determine whether HG004 as gene therapy is safe and effective for the treatment of Leber Congenital Amaurosis caused by mutations…
The objective of this study is to collect long-term safety information (i.e., for 5 years after treatment) associated with voretigene neparvovec-rzyl (vector an…
A recombinant adeno-associated virus serotype 2 (rAAV2) vector has been altered to carry the human RPE65 (hRPE65) gene. This vector has been shown to restore vi…
The objectives of this study are to evaluate the effect of a group psychotherapy intervention for parents of children/adolescents with LCA on indicators of emot…