One-off gene therapy treatment for rare infant disease set to cost over $2 million

A new gene therapy treatment for pediatric patients with spinal muscular atrophy (SMA) has been approved by the U.S. Food and Drug Administration. The single dose treatment, designed to correct a gene mutation causing the rare disease, has been priced at US$2.1 million by pharmaceutical company Novartis, making it the most expensive drug in the world to date.

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Category: Medical

Tags:

• DNA
• Drugs
• FDA
• Genetics

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