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LUXTURNA, made available to European patients by Novartis

DBGenGene therapyLeber Congenital AmaurosisRetinal dystrophyScience and vision
Spark Therapeutics enters into a licensing agreement with the big pharmaceutical company Novartis to commercialize LUXTURNA, the first gene therapy treatment approved for inherited retinal dystrophies, in Europe. LUXTURNA is addressed to patients suffering from Leber Congenital Amaurosis (LCA) caused by mutations in the RPE65 gene. This one-time gene therapy consists of a subretinal microinjection of therapeutic adenoassociated virus that contain a normal copy of RPE65. Corrected retinal...
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DBGen has new website!

DBGenEvents
Starting from January 15 2018 DBGen has a new website. DBGen, which is a company devoted to genetically diagnosing hereditary eye diseases, is a spin-off from the University of Barcelona funded by the researchers Dr Roser González Duarte and Dr Gemma Marfany. They have been conducting basic research on genetic diseases of the retina for over 25 years. DBGen offers genetic...
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LUXTURNA- The first one-time gene therapy treatment for an inherited retinal blindness is approved for use in the US

DBGenGene therapyRetinal dystrophyScience and vision
The FDA (Food and Drug Administration de EEUU) has just approved the first gene therapy treatment for inherited retinal dystrophies that targets a rare disease caused by mutations in a specific gene (precision medicine). After successful clinical trials, FDA has granted LUXTURNA to Spark Therapeutics. With a cost around 850.000 $ (two eyes treatment), LUXTURNA is addressed to patients that carry biallelic...
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