Solving the mysteries of the brain and nervous system to improve health

Rare and ultra-rare diseases affect millions of people worldwide. Today, roughly 10,000 known rare and ultra-rare diseases affect more than 30 million Americans and their families. The incidence of each disease may be rare, but collectively, rare diseases represent a unique public health challenge. Further, rare diseases disproportionately affect the nervous system, and almost 90% of rare disorders can occur in children. For years, the NIH and NINDS have supported important research efforts and partnerships that aim to better understand, diagnose, and treat rare diseases.