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Rare Disease: Could Existing Drugs Turn the Tide?

existing drugs have the potential to make a significant impact on the treatment of rare diseases. Repurposing drugs, which involves using approved or investigational drugs for new indications, can be a promising strategy for rare diseases for several reasons: Known Safety Profiles: Existing drugs have established safety profiles, which can expedite the approval process for new indications. This can be especially beneficial for rare diseases, where developing a completely new drug from scratch can be time-consuming and expensive. Faster Development Timelines: Repurposing drugs can significantly shorten the drug development timeline. Since these drugs have already undergone preclinical and clinical testing for other indications, much of the required safety and efficacy data may already be available. Reduced Costs: Developing a new drug is an expensive process that can cost billions of dollars. Repurposing existing drugs can be a more cost-effective approach, as it bypasses much of the...