… with a genetic condition called Leber congenital amaurosis-4, which meant she could …

… largely targeting orphan conditions like Leber congenital amaurosis 5 (LCA5), bestrophin-1-related …

… patients with a form of Leber congenital amaurosis, according to a press release … patients with a form of Leber congenital amaurosis, according to a press release …

… cause different forms of bestrophinopathy, Leber congenital amaurosis (LCA) and retinitis pigmentosa. The …

… 17. These participants suffer from Leber Congenital Amaurosis (LCA), a blindness caused by … Mark Pennesi, MD, PhD. About Leber Congenital Amaurosis Leber Congenital Amaurosis (LCA) is a severe genetic …

… therapy, under investigation for Leber congenital amaurosis, demonstrated signs of biological … for the treatment of Leber congenital amaurosis, according to a press … administration in patients with Leber congenital amaurosis resulting from biallelic mutations …

… -101, Atsena’s program for Leber congenital amaurosis type 1 (LCA1), one of …

… clinical trial in patients with Leber congenital amaurosis caused by biallelic mutations in … ATSN-101 in patients with Leber congenital amaurosis caused by biallelic mutations … ATSN-101 for GUCY2D-associated Leber congenital amaurosis (LCA1). ATSN-201, which …

… , Luxturna, for the treatment of Leber congenital amaurosis (LCA). CHLA was the first …

… gene therapy in GUCY2D-associated Leber congenital amaurosis. Fifteen patients received unilateral subretinal … gene therapy in GUCY2D-associated Leber congenital amaurosis. Image: Adobe Stock There were …