The promise of gene therapy looms large for families dealing with rare, genetic disorders. Such treatments offer the possibility of one-time cures. But families and researchers worry such therapies will remain out of reach. Collectively, about 350 million people...
Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune deficiency. But it’s not available to him. In 2022, London-based Orchard Therapeutics stopped investing in an experimental treatment for the condition, Wiskott-Aldrich...
MBALE, Uganda (AP) — 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...