Abstract
Rare diseases affect a small percentage of the population but represent a significant unmet medical need. Drug repurposing—the strategy of identifying new therapeutic uses for existing drugs—offers a cost-effective and time-efficient approach to address this challenge. This article reviews the rationale, methodologies, and regulatory pathways associated with drug repurposing. It also highlights successful examples and current initiatives that demonstrate its potential to expedite access to therapies for patients with rare diseases.
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