Understanding the molecular mechanisms of different diseases allows us to develop novel medicines. Yet, developing a brand-new drug is relatively costly and laborious, especially for treating rare diseases. Drug repurposing has been progressively growing in recent decades, which revolutionises the drug development process by shortening the time frame and lowering the investment cost. In fact, drug repurposing may be a promising approach to address the current translational gap in drug development, and alleviate the suffering of millions of patients affected by rare diseases globally.
drug development, drug repurposing, rare diseases, translational gap