ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced that it received Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration (FDA) for sepofarsen for the treatment of Leber’s congenital amaurosis 10 (LCA10), the most common cause of blindness due to genetic disease in children.
October 15, 2019
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