Published 3 months ago

Gene Therapy May Cure Rare Diseases. Drugmakers Have Few Incentives leaving families desperate

Summary by caro.news

(AP)—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. “We feel like we are the forgotten,” said Alderman, who’s advocated for her 21-year-old son since he was a b…

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Gene Therapy May Cure Rare Diseases. Drugmakers Have Few Incentives leaving families desperate

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