Gary Puckrein sees a dilemma coming that has him excited, and at the same time, worried.
In the next year or so, U.S. regulators may approve gene therapies – from Vertex Pharmaceuticals and Bluebird Bio – to treat sickle cell disease, an inherited group of blood disorders that affect an estimated 100,000 Americans. The condition is particularly prevalent among Black people, with 1 of every 365 Black individuals in the U.S. born with the condition, which can cause significant pain, infections, and lead to an early death.
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