Faith Raife has never known a life without hospitals. Born with a criss-cross heart, an extremely rare congenital defect ...
One in 10 Americans live with a rare disease, which often lacks effective treatments. Such is the case for PIGA-CDG, an ultra ...
Researchers at Washington University School of Medicine in St. Louis have conducted a longitudinal study on an individual ...
Gene editing is already being used to treat a handful of rare genetic diseases; however, most therapies involve breaking or ...