Showing 1086 results
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Press release /Luxturna* (voretigene neparvovec) is the first gene therapy to treat an inherited retinal disease, indicated for children and adults with vision loss caused by mutations in both copies of the RPE65…
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Press release /There are no currently approved therapies impacting disease progression for the up to 8 million people suffering from geographic atrophy (GA)1,2 Acquisition will add GT005 to the Novartis portfolio…
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Press release /If approved in expected timeframe, Luxturna* (voretigene neparvovec) will be first and only gene therapy available in EU to treat a rare inherited retinal disease Children and adults…
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Story /These stories offer a glimpse into the lives of people touched by a new era of medicine.
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Press release /Zolgensma® (onasemnogene abeparvovec) is conditionally approved in Europe for the treatment of patients with spinal muscular atrophy (SMA) and a clinical diagnosis of SMA Type 1; or SMA patients with…
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Story /We leave the lab to find out what people know about cell and gene therapy.
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Ad hoc release /One-time treatment with Zolgensma (onasemnogene abeparvovec-xioi) is designed to replace lifetime of chronic therapy for all pediatric patients with SMA Annualized cost of Zolgensma is USD…