MedPage Today June 21, 2024
Associated Press

— Profitability, production cost, and low number of patients are barriers

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 investigational 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 baby.

Collectively, about 350 million people worldwide suffer from rare diseases, most of which are genetic. But each of the 7,000 individual disorders affects perhaps a few in a million people or less. There’s little commercial incentive to develop or bring...

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