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

Robin Alderman faces an agonizing reality: Gene therapy might cure her son Camdens rare, inherited immune deficiency. But its 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.

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Excerpt from:
Gene therapy may cure rare diseases. But drugmakers have few incentives, leaving families desperate - goskagit.com