Featured Studies

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.

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.

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.

If you or someone you know is drinking too much, the National Institutes of Health (NIH) may be able to help. We conduct studies on how alcohol affects the body and are looking for new ways to treat alcohol problems. If you qualify, you can receive alcoholism treatment at no-cost while you participate in our research.

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.

Researchers at the National Institutes of Health (NIH) Clinical Center are testing if spironolactone can be used to treat PAH. Spironolactone is a medication that can help with fluid retention, however it may also be beneficial to adults with PAH by controlling inflammation and improving blood vessel function in the lungs.