mbale, uganda — Barbara Nabulo was one of three girls in her family. But when a sister died, her mother wailed at the funeral that she was left with just one and a half daughters. The half was the ailing Nabulo, who at age 12 understood her mother’s meaning. “I hated myself so much,” Nabulo … Read more
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 said he’s “praying the treatment should come to us.” But experts say the one-time treatment … Read more
The U.S. Food and Drug Administration (FDA) on Friday approved a pair of gene therapies for sickle cell disease, including the first treatment based on the breakthrough CRISPR gene editing technology. The agency approved Lyfgenia from bluebird bio, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals and CRISPR Therapeutics. Both the therapies were … Read more
