Orphan CNS diseases encompass a range of rare neurological disorders that affect the brain and spinal cord, posing significant challenges due to their low prevalence. These conditions often result in limited research and treatment options, leaving affected individuals facing a host of difficulties, including delayed diagnosis, insufficient medical care, and significant impacts on quality of life. Despite their rarity, the collective impact of orphan CNS diseases underscores the need for increased medical research, awareness, and support to address the unique challenges faced by patients and their families. This overview highlights the urgent call for dedicated efforts to improve understanding, treatment, and resources for these underserved conditions.