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  • Conversation: FDA approves gene therapy for a type of blindness

    • December 21, 2017 12:56 PM GMT
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      FDA approves gene therapy for a type of blindness

      The US Food and Drug Administration has approved a gene therapy treatment for patients with a rare inherited eye disease.
      Voretigene neparvovec, which will be sold as Luxturna, is made by Philadelphia-based Spark Therapeutics Inc.
      The one-time treatment is approved for children and adults with retinal dystrophy due to a mutation of the RPE65 gene, which causes severe visual impairment beginning in infancy. As it progresses, patients experience gradual loss of peripheral and central vision, which can eventually lead to blindness.
      There are more than 200 genes that can cause retinal dystrophy. Patients can be tested to determine whether the cause is indeed a mutation of the RPE65 gene and they are thus a potential candidate for this new treatment.
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