For Abigail this will be a specific treatment for her condition: Leber Congenital Amaurosis (LCA) with mutations in the RPE65 gene.
The medicine is called Luxturna (https://luxturna.com/) and one dose should be applied to each eye. Only authorized hospital centers with the necessary training and equipment may apply this treatment. Children's Hospital of Philadelphia (CHOP) in the United States is one of them. In that hospital, Abigail's case was evaluated in October 2019 and they gave positive results for said treatment.
This is a treatment for patients who have not yet totally lost their vision, LUXTURNA will prevent the death of photoreceptors, allowing the preservation of visual acuity.
Apart from being one of the first genetic therapies that are available for treatment, Luxturna is a very expensive drug because it is an orphan drug and very new, that is, this treatment is for a rare disease suffered by very few people in the world and therefore its price is so high.
Its application in each eye has a value of US $ 425,000, for both eyes US $ 850,000, plus hospital and transportation expenses.