Drug approved as sickle cell disease treatment may help stabilize vision in rare genetic disease

Ophthalmologist Rajendra S. Apte, MD, Ph.D., (right), of Washington University School of Medicine in St. Louis, examines patient Patricia Collins (left) while medical student Wilson Wang observes. Collins is a participant in a clinical trial that tests the safety and efficacy of an FDA-approved drug in stabilizing vision in patients with RVCL-S, a rare genetic disease that affects tiny blood vessels in the body. Credit: Matt Miller Roughly 50 families scattered across the world share ultra-rare variants in a particular gene. Silent for years, the inherited mutations make themselves known Read More