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.

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 are conducting a research study to learn more about the motor skill and brain development of infants who are at high-risk for or diagnosed with Cerebral Palsy (CP) and Autism Spectrum Disorder (ASD) compared to infants with typical development. The results of this study may assist with better methods for early diagnosis as well as improved treatment for children with CP and ASD. The results of this study may eventually assist with better methods for early diagnosis as well as improved treatment for children with autism.

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.

Gigantism is a condition characterized by excessive growth and height significantly above average, caused by over-production of growth hormone (GH) during childhood. Investigators at the National Institutes of Health (NIH) want to see if a drug that antagonizes growth hormone action, called pegvisomant, can help children and adolescents with gigantism.

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.