Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate
https://apnews.com/article/rare-disease-wiskott-aldrich-genetics-gene-therapy-1f3aded94c30eba83367eb4e2c0d61e6
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 syndrome. And there are no gene therapy studies he can join.
