Latest Gene therapy News

Takeaways from AP's report on access to gene therapies for rare diseases

Jun. 21, 2024 00:43 AM EDT

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...

Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate

Jun. 21, 2024 00:39 AM EDT

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...

A rural Ugandan community is a hot spot for sickle cell disease. But one patient gives hope

May. 12, 2024 02:08 AM EDT

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...