More News! 11 Oct 2019
RNA Therapy Improves Vision in Untreatable Genetic Blindness
A phase I/II trial run by the Dutch company ProQR has found that its RNA therapy could significantly improve the vision of people with Leber’s congenital amaurosis, a rare genetic disease for which there is no treatment. The RNA drug, called sepofarsen, is designed to treat people with a specific mutation in a gene […]