Lifesaving gene therapy for kids is world's priciest drug at $4.25M
Briefly

In an initial trial, 37 children who received the gene therapy were all still alive at age 6, most could talk, walk, and perform well on IQ tests.
The price set by Lenmeldy for the US market exceeds experts' calculations for a fair price, potentially causing significant challenges for healthcare systems.
Read at Ars Technica
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