STAT+: Cutting-edge gene therapies could be coming for sickle cell. Will Medicaid patients be able to access them?
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 – one developed by Vertex Pharmaceuticals, and another by 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.
