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

The promise of gene therapy looms large for families dealing with rare, genetic disorders. Such treatments…

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

Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camden’s rare, inherited immune…

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

MBALE, Uganda — Barbara Nabulo was one of three girls in her family. But when a…