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Story /Novartis is taking its investigational gene therapy for a degenerative eye disease into clinical trials.
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Story /Dr. Botond Roska says we should keep our eyes on gene therapy.
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Story /Novartis and Sangamo Therapeutics plan to pursue potential treatments for neurodevelopmental diseases.
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Ad hoc release /SMA is a rare genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, permanent ventilation or death for most patients by age 2[1],[2…
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Ad hoc release /AveXis lead product candidate, AVXS-101, has potential to be first-ever one-time gene replacement therapy for spinal muscular atrophy (SMA), a disease which results in early death or lifelong…
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Press release /Facility to become the largest of four state-of-the-art sites involved in manufacturing of AveXis gene therapies for pipeline of rare genetic diseases including spinal muscular atrophy …
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