I heard a fascinating story from NPR last week. The Federal Drug Administration recently approved a new gene therapy that has a whopping $2.125 million dollar price tag. It’s the most expensive drug on the market.
The gene therapy is a treatment for a rare disease called Spinal Muscular Atrophy (SMA). According to the NPR story, SMA is “a rare disorder caused by a defective gene; the illness destroys the nerves that control muscles. Babies with the most severe form of the disorder typically don’t live past their second birthday.”