National Institute of Neurological Disorders and Stroke (NINDS)

Rare Disease Research at NINDS

When most people think of neurological disorders, a small number of diseases that affect millions of people—such as epilepsy, Parkinson’s disease, and stroke—come to mind. While we focus a majority of our effort on these devastating diseases, we also have very dedicated research programs devoted to finding treatments for numerous rare diseases, which are defined as conditions affecting fewer than 200,000 Americans. In fact, due to the prevalence of neurological symptoms in rare diseases—of the estimated 7,000 known rare diseases that lead to significant morbidity and mortality in 25 million people in the United States, a third are thought to include a neurological component—NINDS funds research in to an extraordinary number of rare diseases.

Examples of rare neurological conditions that NINDS-funded scientists and clinicians study are amyotrophic lateral sclerosis (ALS), Duchenne muscular dystrophy, and Huntington’s disease. In addition, NINDS funded research of hundreds of other rare diseases, including cerebrovascular diseases, neurometabolic disorders, neuromuscular and neurodegenerative disorders, movement disorders, epilepsies... More Message »