Featured Studies

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.

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.

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.

Researchers at the National Institutes of Health (NIH) are testing NX-2127, an oral drug that degrades a protein in cancer cells called Bruton's tyrosine kinase (BTK), instead of inhibiting or blocking it like other drugs. NX-2127 may also stimulate your immune T-cells to attack the cancer.