Gene therapy for Leber congenital amaurosis showed evidence of safety and possible efficacy in two preliminary studies. Leber congenital amaurosis (not to be confused with Leber optic neuropathy ...

MeiraGTx's financials show $125 million in cash, but additional funding will be needed within the next 12 months. Click for ...

Disruption of photoreceptor cilia has been implicated in a variety of retinal diseases, collectively called retinociliopathies, such as Leber congenital amaurosis and retinopathy of prematurity.

US genetic meds company MeiraGTx Holdings yesterday revealed that the US Food and Drug Administration (FDA) has granted the company Rare Pediatric Disease (RPD) designation to its AAV8-RK-RetGC ...

People with this form of Leber congenital amaurosis (LCA1 ... who also co-directs the Center for Hereditary Retinal Degenerations at UPenn. "One patient reported for the first time being able ...

MeiraGTx (MGTX) announced the U.S. Food and Drug Administration, FDA, has granted the Company Rare Pediatric Disease Designation, RPDD, to its ...