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  • Gene therapy

    Gene therapy works by replacing the defective mutated gene in the affected cells with a normal healthy copy, primarily in inherited retinal disorders.
  • Leber congenital amaurosis (LCA)/Early onset severe retinal dystrophy (EOSRD): for professionals

    LCA/EOSRD form part of a spectrum of inherited retinal dystrophies that cause severe visual loss at an early age. There is now an approved retinal gene therapy called Luxturna for patients with RPE65 mutations.
  • RPE65 gene

  • Leber Congenital Amaurosis (LCA): for patients

    LCA describes a group of inherited retinal conditions that cause severe sight impairment or blindness from birth. There is now an approved treatment called Luxturna for patients with RPE65 mutations.
  • Luxturna treatment in the UK

    Treatment centres in the UK currently offering Luxturna treatment under the NHS.

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