MeiraGTx Receives Rare Pediatric Disease Designation from FDA for AAV8-RK-RetGC for the Treatment of Patients with Leber Congenital Amaurosis due to GUCY2D Mutations
(NASDAQ:MGTX), LONDON and NEW YORK, Jan. 22, 2025 (GLOBE NEWSWIRE) — MeiraGTx Holdings Plc (Nasdaq: MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the U.S. Food and Drug Administration (FDA) has granted the Company Rare Pediatric Disease Designation to its AAV8-RK-RetGC program for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations […]