Current Drug Repurposing Strategies for Rare Neurodegenerative Disorders.

ABSTRACT: Rare diseases are life-threatening or chronically debilitating low-prevalent disorders caused by pathogenic mutations or particular environmental insults. Due to their high complexity and low frequency, important gaps still exist in their prevention, diagnosis, and treatment. Since new drug discovery is a very costly and time-consuming process, leading pharmaceutical companies show relatively low interest in orphan drug research and development due to the high cost of investments compared to the low market return of the product. Drug repurposing-based approaches appear then as cost- and time-saving strategies for the development of therapeutic opportunities for rare diseases. In this article, we discuss the scientific, regulatory, and economic aspects of the development of repurposed drugs

SUBMITTER: Shah S 

PROVIDER: S-EPMC8724568 | biostudies-literature | 2021

REPOSITORIES: biostudies-literature