A recent gene therapy trial in Leander, Texas, has sparked debate among experts and families affected by rare diseases. Andrew Revell, a young patient with a debilitating muscle disorder, participated in the clinical trial for over a year but remains skeptical about its effectiveness.
According to Louise Rodino-Klapac, President of Research and Development at an organization focused on rare diseases, the treatment’s ambiguous data raises concerns about its safety and efficacy. The high costs associated with gene therapies also fuel worries about pharmaceutical companies’ exploitation of vulnerable families.
Experts emphasize the need for rigorous scientific evidence before approving such treatments. A recent guest column by cardiologist Eric Topol highlights 10 science-based ways to prevent heart disease, shifting attention away from the Epstein scandal dominating headlines. Meanwhile, a comment piece by Ian Duncan questions the effectiveness of “college hazing” or training programs in addressing air traffic recruitment issues.
As the debate surrounding gene therapies continues, families like Revell’s urge policymakers to address the root causes of these diseases rather than just treating their symptoms. AI experts Youyou Zhou note that emerging technologies may impact 700 professions, raising questions about job security and the future of work.
Source: https://www.washingtonpost.com/business/2025/07/26/fda-rare-disease-sarepta-duchenne