Featured Studies

Myelodysplastic syndromes (MDS) are disorders caused by abnormal blood cell production, potentially leading to low cell counts and increased susceptibility to clotting and infections. Past research suggests that inflammation may trigger these abnormalities, and this study seeks to further understand and improve treatment strategies for MDS by investigating the role of inflammation in bone marrow cell development.

Doctors at the National Institutes of Health (NIH) are conducting a research study to determine the viability and safety of early initiation of oral therapy with cyclosporine and eltrombopag in patients with severe aplastic anemia (SAA).

Researchers at NIH are testing a new therapy in patients diagnosed with colitis due to chronic granulomatous disease (CGD). The research aims to see if restoring the gut microbiome reduces inflammation in the colon.

We are looking for people recently diagnosed with Lyme disease to help us answer important questions about the infection. If you have or had the rash associated with acute Lyme disease (erythema migrans), facial palsy, meningitis, heart block, or arthritis; please consider participating in our study.

Do you have Parkinson's disease? Doctors at the National Institute of Neurological Disorders and Stroke (NINDS) are conducting a research study on people with Movement Disorders. They are currently recruiting participants with Parkinson's disease. The purpose is to learn more about the disease and identify genetic changes and other indicators of the condition that may allow earlier diagnosis.There is no cost for study-related tests or procedures. Travel assistance may be provided.

The National Institute of Neurological Disorders and Stroke at the National Institutes of Health Clinical Center is looking for individuals with severe epilepsy or individuals who have experienced persistent seizure activity that cannot be controlled with medication, to participate in this research study. The goal of this research study is to provide surgical treatment to control these types of seizures. Participation requires an inpatient stay of up to 4 weeks, plus 3 outpatient visits within 2 years after surgery.

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.

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.