Abstract
Rare genetic disorders affect a small portion of the population but collectively pose significant healthcare challenges. Pharmacological advancements have introduced targeted therapies that address the molecular and genetic basis of these disorders, leading to improved outcomes and quality of life. This article discusses the current pharmacological approaches to treating rare genetic diseases, including enzyme replacement therapy, gene-based therapy, and small molecule drugs. It also explores the challenges in drug development, regulatory incentives, and future prospects for precision medicine in this domain.
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