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Translational Science at the NIH

Philip John Brooks, PhD, Program Director, Office of Rare Diseases Research, National Center for Advancing Translational Sciences, National Institutes of Health (NIH) discusses his work in rare diseases at the NIH. Dr. Brooks is focused on accelerating clinical trials in rare diseases by moving beyond “one disease at a time” approaches. Examples include the development of therapeutics that target shared molecular mechanisms underlying multiple rare diseases, and the implementation of platform vector gene therapy trials.