Gene therapy could treat rare diseases. But drugmakers have little incentive, leaving families helpless
Robin Alderman faces a painful truth: Gene therapy can cure her son Camden’s rare, inherited immune deficiency. But this is not available to him. London-based Orchard Therapeutics stopped investing in an experimental treatment for Wiskott-Aldrich syndrome in 2022. And there are no gene therapy studies for him to participate in. “We feel like we’ve been… Read More »