
Eltrombopag for People With Fanconi Anemia
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.

Unrelated Umbilical Cord Blood Transplantation for Severe Aplastic Anemia and Hypo-plastic MDS using CordIn(TM), Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells to Expedite Engraftment and Improve Transplant Outcome
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.

Natural History and Genetics of Food Allergy and Related Conditions

Children's Growth and Behavior Study

Impact of Elastin Mediated Vascular Stiffness on End Organs
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.

Impact of Elastin Mediated Vascular Stiffness on End Organs
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.

Clinical and Scientific Assessment of Pain and Painful Disorders
Researchers in the National Center for Complementary and Integrative Health at the National Institutes of Health seek healthy volunteers to participate in a study investigating how our brain responds to pain. This study will allow researchers to collect information that may be used to learn more about pain disorders and how we respond to painful experiences.

Studies of the Natural History, Pathogenesis, and Outcome of Idiopathic Systemic Vasculitis
The National Institute of Arthritis and Musculoskeletal and Skin Diseases at the National Institutes of Health (NIH) in Bethesda, Maryland is conducting a study for vasculitis. Researchers are inviting both volunteers who have the condition, and those who do not, to participate in this study. All study-related tests and procedures are provided at no cost to you.