Featured Studies

Researchers at the National Institutes of Health (NIH) seek patients with Williams Syndrome (WS) or Supravalvular Aortic Stenosis (SVAS) to join a research study. Researchers want to see how blood vessel elasticity and stiffness affect the heart, gut, kidneys, and the brain in people with Williams Syndrome.

Multiple sclerosis (MS) is a disease that damages the central nervous system (brain and spinal cord). This leads to increased physical disability over time. The disease is lifelong once it begins. Researchers want to learn more about the stages of multiple sclerosis and follow them until a person's death to understand how the physical and clinical signs of MS relate to its changes over time.

The National Institutes of Health (NIH) is looking for volunteers who use opiates regularly or who are receiving treatment for opiate use. The purpose is to learn how opiates affect brain function.

National Cancer Institute (NCI) researchers are conducting a study of patients with rare central nervous system (CNS) tumors. More than 130 primary rare CNS (brain or spine) tumors have been identified. Most affect less than 2,000 people in the United States each year. This study will test whether stimulating the immune system using the drug nivolumab can shrink select rare brain or spine tumors or increase the time it takes for them to grow or spread.

Women diagnosed with polycystic ovary syndrome (PCOS) needed for a research study at the National Institutes of Health (NIH). Researchers want to learn more about how a medication may be used to help your body's insulin work better and if this can improve your overall metabolic health.

Severe aplastic anemia is a rare and serious blood disorder. Patients with severe aplastic anemia commonly receive Cyclosporine to help improve blood counts. When cyclosporine treatment stops, the disease may return in one in three people. Researchers at the National Institutes of Health (NIH) Clinical Center are studying if sirolimus, an immunosuppressant, can help prevent a relapse in patients diagnosed with severe aplastic anemia following cyclosporine treatment.

Treatment of severe aplastic anemia (SAA) and myelodysplastic syndrome (MDS) usually involves a bone marrow transplant. Researchers at the National Institutes of Health (NIH) are testing ways to make transplant using unrelated donor cells from umbilical cord blood safer and more effective. We hope these procedures will reduce post-transplant complications and improve transplant outcomes.

Fanconi anemia is a rare genetic disorder that mostly affects children and often leads to bone marrow failure. Researchers at the National Institutes of Health (NIH) are investigating a new drug to improve blood counts in individuals diagnosed with Fanconi anemia. Eltrombopag is an oral drug that mimics a special protein that causes the body to make more platelets. Currently eltrombopag is used to treat low platelet counts in patients with hepatitis C and chronic immune thrombocytopenic purpura (ITP). Researchers are evaluating if this new drug will improve blood cell counts in individuals diagnosed with Fanconi anemia.