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 grasped her mother’s meaning. “I hated myself so much,” Nabulo … Read more
A young patient died due to cardiac arrest after receiving Pfizer’s experimental gene therapy being tested in a mid-stage trial for a muscle-wasting disorder called Duchenne muscular dystrophy(DMD), the drugmaker told Reuters on Tuesday. “A fatal serious adverse event was reported as cardiac arrest for a participant in the Phase 2 DAYLIGHT study,” a company … 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 BLUE.O, and a separate treatment called Casgevy by partners Vertex Pharmaceuticals VRTX.O and CRISPR Therapeutics CRSP.BN. Both the therapies were approved for people … Read more
Regulators on Friday approved two gene therapies for sickle cell disease that doctors hope can cure the painful, inherited blood disorder that afflicts mostly Black people in the U.S. The Food and Drug Administration said the one-time treatments can be used for patients 12 and older with severe forms of the disease. One, made by … Read more
