FDA approved a drug to treat a rare and fatal genetic disease in children, a life-saving therapy that will cost $2.1M.
They don't know if their children will ever walk. But finding other families online has given them hope.
She remembers watching a video for one the trials that showed the impact of a single dose of gene therapy in pigs. Initially the pigs were shown dragging their feet, but after therapy, “it show[ed] the pig walking perfectly,” Almeida said. “We knew right away that this was it. This was what we wanted for Matt.”
“Critics are not actually thinking about how our healthcare systems work,” Vas Narasimhan said. “For transplants, we spend $3 million to $5 million [per patient] with far less efficacy than we see with a medicine like this. We’re trying to price this at 50 percent of what is the current cost of care over ten years for these children.”
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