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The Independent UK
The Independent UK
National
Laura Ungar

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

Gene Therapy Disease Orchard Claire Booth Donald Kohn Estella United States
Copyright 2024 The Associated Press. All rights reserved.

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.

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