Katie Jackson desperately wants a new treatment for Huntington’s disease. Her husband died from the devastating brain disorder. And because the disease runs in families, her three children have a 50% chance of inheriting the condition. She’s pinned her hopes on a cutting-edge gene therapy from
But Jackson says Huntington’s patients have no desire to meet a new demand from the US Food and Drug Administration: to enroll in a new clinical trial where some people will undergo fake brain surgery without getting UniQure’s treatment. The FDA’s request, intended to conclusively determine the therapy’s potency, has ...