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However, since then, the field has continued to progress rapidly, with recent animal studies and early-phase clinical trials that highlight the efficacy and feasibility of IN delivery for iron chelators ( 2 Impaired iron homeostasis and brain dysfunction Iron is essential for multiple biological functions, including oxygen transport, redox actions, and DNA synthesis (Khn, 2015)

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A key point in the small-molecule discovery process that could provide opportunities to help address this challenge is the pharmacological characterization of hit and lead compounds, culminating in the selection of a drug candidate