Rare Diseases

Rare diseases, also known as orphan diseases, are medical conditions that affect a small percentage of the population. Typically, a disease is classified as rare when it affects fewer than 200,000 individuals in the United States or a similar low prevalence in other countries. These diseases can vary widely in their nature, severity, and symptoms, and many have genetic origins. Due to their rarity, often there is limited research, treatment options, and support for individuals affected, which can lead to challenges in diagnosis and management. Rare diseases encompass a diverse range of conditions, including certain types of cancers, neurological disorders, and metabolic syndromes. Efforts to address the needs of patients with rare diseases include advocacy for increased research funding, development of therapies, and the establishment of support networks.