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This section presents essays on human studies of early-onset familial AD (eFAD). Families with this condition have been excluded from drug studies, even though the genes causing disease in their families have done much to advance AD research in the past 15 years. Why is that? The essays in this section aim to illuminate some of the complex issues of drug research in AD that make it challenging to design clinical trials for eFAD. In addition, we aim to articulate how eFAD families hold out the tantalizing prospect of moving drug research toward prevention of this disease, and what kind of observational clinical research could set the stage for eventual prevention studies.