Advances in orphan drugs for the treatment of rare nervous system diseases

Abstract:

Rare diseases are characterized by a low prevalence rate，unknown etiology，severe symptoms，and poor treatment outcomes. Orphan drugs are developed for the prevention，treatment，and diagnosis of these rare diseases. Due to the limited number of patients with rare diseases and the limited research funds in enterprises，these drugs are often scarce and expensive. However，with the improvement in the public awareness of rare diseases，the introduction of incentive policies，and the increase in research investments in recent years，rapid progress has been made in the research and development of orphan drugs，with a significant increase in the proportion of orphan drugs for the treatment of tumors，pediatric diseases，and nervous system diseases. Many rare diseases affect the nervous and muscular systems，and therefore，this article reviews the drugs for the treatment of rare nervous system diseases.