gene therapy
Gene therapy uses molecular tools to change the genetic code in cells and correct DNA mutations with the goal of curing diseases, including cancers and inherited genetic disorders such as hemophilia and sickle cell disease. Here you can learn about how it works, the companies developing gene therapies, and the debate about the cost of these treatments.
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‘That’s $425,000 right there’ — the anxious launch of a gene therapy with a record sticker price
Should employers take a chance on little-known Embarc to pay for gene therapy?
30 years is too long to wait for new medicines. There are ways to speed up drug development
STAT Plus: Uniqure’s Huntington’s gene therapy lowers key protein in small trial
STAT Plus: FDA plans to speed path to approval for some gene therapies, starting with hemophilia
There’s a possible cure for ‘bubble boy’ disease. It will cost $665,000
STAT Plus: FDA’s Califf talks cell and gene therapies, abortion pill, Alzheimer’s drugs
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