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inherited retinal disease

This page shows the latest inherited retinal disease news and features for those working in and with pharma, biotech and healthcare.

Novartis negotiates ex-US rights to Spark gene therapy

Luxturna is the first gene therapy to be approved in the US where it has a similar indication - biallelic RPE65-mediated inherited retinal disease (IRD) - and is being launched at a ... No otherwise healthy child should have to go blind due to this

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Spark sets Luxturna price, igniting US affordability debate

Luxturna is used to treat a sight-robbing disorder called biallelic RPE65-mediated inherited retinal disease (IRD), which affects somewhere between 1, 000 and 2, 000 people in the US with
Spark’s Luxturna becomes US’s first approved gene therapy

The FDA has chalked up another first for 2017, approving Spark Therapeutics’gene therapy Luxturna for a sight-robbing disorder called biallelic RPE65-mediated inherited retinal disease (IRD). ... a policy framework - including the development of disease
Spark closes on first US gene therapy approval

Spark closes on first US gene therapy approval. FDA starts review of Luxturna for patients with inherited retinal disease. ... Luxturna (voretigene neparvovec) is under review for a condition known as biallelic RPE65-mediated inherited retinal disease

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