How do the 12 FDA-approved gene therapies break down by medical indication

The 12 FDA-approved gene therapies cover a range of medical indications, primarily focusing on genetic disorders, cancers, and hematological conditions. Here’s a breakdown of these therapies by their medical indications: Genetic Disorders: Luxturna (voretigene neparvovec-rzyl): Approved for the treatment of patients with confirmed biallelic RPE65 mutation-associated retinal dystrophy, which leads to vision loss and may […]

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Revolutionizing Public Health: CBER’s Peter Marks and the Vision to Reduce FDA Visits

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