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 ...
A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic neuropathy (LHON). The study was conducted in collaboration with the Seoul National ...
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 ...
A rare genetic disease left a Coronado teenager legally blind at 13. Now he's running track — and helping drive research for ...
A research team has successfully demonstrated the world's first gene-editing treatment for Leber's hereditary optic neuropathy (LHON). The study was conducted in collaboration with the Seoul National ...
A Prescription Drug User Fee Act target date of February 28, 2026 has been set for the application. The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for ...
Researchers from Landeskrankenhaus Salzburg (SALK) - Universitätsklinikum der PMU have identified a novel loss-of-function variant in the NDUFA7 gene in a patient with Leber’s hereditary optic ...
MeiraGTx has recently received Rare Pediatric Disease Designation (RPDD) for four inherited retinal diseases (IRDs) reflecting the transformative therapeutic potential of the Company’s proprietary ...
LONDON and NEW YORK, Jan. 22, 2025 (GLOBE NEWSWIRE) -- MeiraGTx Holdings Plc (MGTX), a vertically integrated, clinical-stage genetic medicines company, today announced the U.S. Food and Drug ...
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