The gene therapy Zolgensma, created by Novartis, is used to treat infants and children with spinal muscular atrophy (SMA). A single treatment is available for $2.1 million.
Luxturna: This gene therapy, created by Spark Therapeutics, is used to treat inherited retinal disorders brought on by RPE65 gene mutations. The price for a single treatment is $850,000.
Actimmune: This medication was created by Horizon Therapeutics and is used to treat malignant osteopetrosis and chronic granulomatous disease. The cost has been set at $470,000 annually.
Hemlibra is a medication made by Roche that uses inhibitors to treat hemophilia A. The cost has been set at $380,000 annually.
Ravicti: This medication was created by Horizon Therapeutics and is employed to treat urea cycle disorders. The cost has been set at $350,000 annually.
Kalydeco: This medication, created by Vertex Pharmaceuticals, treats cystic fibrosis brought on by particular genetic mutations. The cost has been set at $311,000 annually.
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