Featured Studies

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.

The research study sets out to learn about changes in the genetic makeup of CLL during the early phase of treatment with venetoclax. The initial phase of venetoclax therapy can be medically and logistically challenging. Patients will receive expert medical care at the National Institutes of Health (NIH).

African-American women, ages 25-75, who are obese or overweight and pre-diabetic, are needed to participate in a research study at the National Institutes of Health (NIH) Clinical Center in Bethesda, Maryland. Researchers want to explore if the use of mobile technology and coaching can help increase physical activity.

Doctors at the National Institutes of Health (NIH) are conducting a study on patients with clonal hematopoiesis of indeterminate potential (CHIP). Patients with CHIP have a genetic mutation that has been associated with blood cancer. The study will look at CHIP and try to determine its relationship to blood cancers and heart disease. The study will also try to discover other new organs or diseases that may be linked with.

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.

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.

Doctors at the National Institutes of Health (NIH) are enrolling people with LAM into a research study. LAM is a rare progressive lung disease that usually strikes women during their childbearing years, between the onset of puberty and menopause. One possible treatment to improve breathing in patients with LAM is inhaled albuterol. Albuterol can be given using a hand held inhaler or with a nebulizer. The study wants to compare these methods to see which method improves lung function in patients with LAM.