Patients with a rare inherited eye disorder that causes vision loss and blindness are being treated with a new form of gene therapy. Luxturna (voretigene neparvovec) is a drug from Novartis that carries a steep price—£600,000. However, NHS England has negotiated a lower price with the manufacturer.
The therapy is for patients who have retinal dystrophy; it’s the result of inheriting a faulty copy of the RPE65 gene. The Luturna injection can stop disease progression and may even improve vision.
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“Gene therapy to halt rare form of sight loss”