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The revolutionary sickle cell therapies
Editor’s Note: Elizabeth Yuko, Ph.D., is a bioethicist and writer whose work has appeared in The New York Times, Rolling Stone, The Washington Post, The Atlantic and elsewhere. She is also an adjunct professor at Fordham University. The views expressed here are hers. Read more opinion on CNN. In December, the US Food and Drug Administration (FDA) approved the first […]
Sickle cell affects more families in Africa and India, but new gene therapies are out of reach
Gautam Dongre’s two children in India and Pascazia Mazeze’s son in Tanzania live with an inherited blood disorder that turns blood cells into instruments of pain. Now that new gene therapies promise a cure for their sickle cell disease, Dongre says he’s “praying the treatment should come to us.” But experts say the one-time treatment […]
US regulators approve two gene therapies for sickle cell disease | US news
The US Food and Drug Administration has approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough Crispr gene-editing technology, opening up two “transformative therapy” avenues for some patients. The FDA approved Lyfgenia from Bluebird Bio, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals and […]