Журнал фармацевтических наук и новых лекарств

Drug Repurposing for Rare Genetic Disorders: Expanding Treatment Options for Orphan Diseases

Yuxin Wang

Rare genetic disorders, also known as orphan diseases, pose significant challenges in terms of diagnosis and treatment. These conditions often lack approved therapies, leaving patients with limited or no treatment options. Drug repurposing, the process of identifying new uses for existing drugs, offers a promising approach to address unmet medical needs in rare genetic disorders. This brief note explores the concept of drug repurposing in the context of rare genetic disorders, highlighting its potential to expand treatment options and improve the lives of patients affected by these conditions.