Please provide your email address to receive an email when new articles are posted on . Opus Genetics received rare pediatric disease designation from the FDA for its ocular gene therapy designed to ...
MeiraGTx receives FDA Rare Pediatric Disease Designation for AAV8-RK-RetGC to treat Leber congenital amaurosis due to GUCY2D mutations. MeiraGTx Holdings Plc announced that the FDA has granted its ...
Two people with a rare inherited eye disorder have had their night vision restored by an experimental gene therapy, researchers say. These two individuals are part of an ongoing clinical trial testing ...