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  1. luxturna.comLUXTURNA® (voretigene neparvovec-rzyl) - Inherited Retinal...

    LUXTURNA is a prescription gene therapy product for patients with RPE65 gene mutations and enough retinal cells. Learn about the indication, safety information, patient support, and treatment journey of LUXTURNA.

    • Take your first step

      Take your first step - LUXTURNA® (voretigene...

    • Patient resources

      Patient resources - LUXTURNA® (voretigene neparvovec-rzyl) -...

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      Sign Up - LUXTURNA® (voretigene neparvovec-rzyl) - Inherited...

  2. luxturna.com › about-luxturnaWhat is LUXTURNA? - LUXTURNA® (voretigene neparvovec-rzyl)

    LUXTURNA is a one-time gene therapy that may improve functional vision in patients with RPE65 gene mutations, a cause of inherited retinal disease (IRD). Learn about the indication, side effects, and how to get genetic testing for LUXTURNA.

  3. en.wikipedia.org › wiki › Voretigene_neparvovecVoretigene neparvovec - Wikipedia

    Voretigene neparvovec, sold under the brand name Luxturna, is a gene therapy medication for the treatment of Leber congenital amaurosis. [4] Leber's congenital amaurosis, or biallelic RPE65 -mediated inherited retinal disease, is an inherited disorder causing progressive blindness.

  4. www.fda.gov › news-events › press-announcementsFDA approves novel gene therapy to treat patients with a rare...

    Luxturna is the first gene therapy in the U.S. that targets a disease caused by mutations in a specific gene. It delivers a normal copy of the RPE65 gene to retinal cells to restore vision in patients with biallelic RPE65 mutation-associated retinal dystrophy.

  5. www.ema.europa.eu › en › medicinesLuxturna - European Medicines Agency (EMA)

    Luxturna is a medicine that is used to treat adults and children with loss of vision due to inherited retinal dystrophy, a rare genetic disorder of the retina (the light sensitive membrane at the back of the eye).

  6. www.fda.gov › vaccines-blood-biologics › cellular-gene-therapy-productsLUXTURNA | FDA - U.S. Food and Drug Administration

    8 Ιουν 2022 · LUXTURNA is a novel gene therapy that treats patients with a rare form of inherited vision loss caused by biallelic RPE65 mutation. It is an adeno-associated virus vector-based product that must be administered to patients with viable retinal cells.

  7. luxturna.com › how-does-luxturna-workHow does LUXTURNA work? - LUXTURNA® (voretigene neparvovec-rzyl)

    LUXTURNA delivers a working RPE65 gene to the retina of the eye to restore the visual cycle and improve functional vision. Learn how LUXTURNA works, how it is administered, and what side effects it may cause.

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