Gene therapy can cure rare diseases. But drug makers have little incentive, leaving families desperate
Robin Alderman faces a painful reality: gene therapy could cure her son Camden’s rare hereditary immune deficiency. But it’s not for 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 participate in. “We feel like we’ve been forgotten,” … Read more