A recent Wall Street Journal column suggested that the U.S. Food and Drug Administration (FDA) is hindering hope for rare disease patients by allowing them to try innovative therapies. However, this right can be hijacked by investors with financial incentives to label treatments as safe and effective based on partial data. The gene therapy AMT-130, currently under development to treat Huntington’s disease, suggests that it may slow its progression by 75% but this claim has serious issues. The FDA does not demand a sham procedure for every procedure and has issued guidance for sham procedures in neurodegenerative disease as highly variable due to the nature of these illnesses. The author argues that the FDA's role is to present a non-distorted benefit and risk analysis and ensure that no company can exploit vulnerable patients.