The video shows one subject that participated in a Phase 1/2 clinical trial of sepofarsen, an experimental RNA therapy for Leber’s congenital amaurosis 10. At the first trial visit, prior to receiving treatment with sepofarsen, the subject performs the mobility course test. The subject is unable to complete a simple course with a straight path and brightly lid high-contrast obstacles. At the end of the trial, at 12 months and having had 3 doses of sepofarsen, he performs the mobility course test again. The subject is now able to complete a much more complex course with various turns and multiple unlit obstacles in a dimly lid room.
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