Featured Studies

Researchers in the National Center for Complementary and Integrative Health at the National Institutes of Health seek healthy volunteers to participate in a study investigating how our brain responds to pain. This study will allow researchers to collect information that may be used to learn more about pain disorders and how we respond to painful experiences.

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.

The National Institutes of Health (NIH) seeks healthy volunteers for a study about blood vessel stiffness. In this study, healthy volunteers will be compared with patients with rare genetic conditions that affect the blood vessels. All study-related procedures are provided at no cost and compensation is provided.

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.

Researchers at the National Institutes of Health (NIH) are studying if the medication danazol can be used to treat people with short telomere disease who also have bone marrow failure, liver, or lung disease. In recent studies, danazol, at high doses, showed a positive influence on telomere length. Additional research is needed to learn more about the role of different doses of danazol on telomere disease as well as its effects on secondary diseases.

Are you from Mexico, Central or South America, or India with hydrocephalus, seizures, or an inflammatory condition of the central nervous system without a diagnosis? The National Institutes of Health (NIH) is studying people with cysticercosis and those likely to have cysticercosis. All study related tests and procedures are provided at no cost to you. Travel to and from NIH may be reimbursed.

The National Institutes of Health (NIH) is conducting a research study for patients with lupus. Doctors want to learn more about the disease and are looking for what causes it, how it progresses over time, and the genetic factors that might make a person more likely to develop it. This study is open to any individual, 10 years of age or older, who meets the American College of Rheumatology's criteria for having lupus, which include symptoms and abnormal blood tests.

Doctors at the National Institutes of Health (NIH) are investigating new techniques needed to develop gene therapy or other treatment for certain inherited immune system diseases. If you have an inherited disease that is affecting your blood or bone marrow cell that generate blood cells, you may be eligible to participate in this research study. Participants will be screened with a medical history, physical examination and blood and urine tests.

Cystic fibrosis (CF) is a progressive, genetic disease that causes persistent lung infections and over time limits the ability of individuals to breathe. Researchers at the National Institutes of Health (NIH) are studying the infection, disease progression, and abnormal genes responsible for cystic fibrosis to better understand the disease process and its effects on breathing.