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95% of Rare Diseases Lack Treatments: New Models Offer Hope

1/10/2025

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Summary

Story summary

Rare diseases impact nearly 10% of people worldwide, but a staggering 95% remain without approved treatments. With over 70% being genetic, the need for tailored preclinical models is urgent. Innovative complex in vitro models, such as iPSCs and organoids, are paving the way for breakthroughs in drug efficacy and clinical trials, urging researchers globally to embrace these advancements.

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Story summary

Rare diseases impact nearly 10% of people worldwide, but a staggering 95% remain without approved treatments. With over 70% being genetic, the need for tailored preclinical models is urgent. Innovative complex in vitro models, such as iPSCs and organoids, are paving the way for breakthroughs in drug efficacy and clinical trials, urging researchers globally to embrace these advancements.