Clinical Trials

Do you have short telomeres and a telomere gene mutation? Some people who have aplastic anemia have very short telomeres, which protect the ends of DNA in chromosomes. This study is testing whether low doses of the medicine danazol help prevent telomeres from getting shorter and reduce signs of damage from aplastic anemia or related conditions. Participants in this study must be 3 years or older and have a telomere disease and signs of aplastic anemia, lung disease such as pulmonary fibrosis, or liver disease. This study is located in Bethesda, Maryland.

Do you or your child have Friedrich's Ataxia? This study is looking into a supplement in the form of vitamin B3 plus exercise can help the body better use oxygen for people with Friedrich’s Ataxia. You must be 10 to 40 years old to join. Participants may be asked to take the daily supplement, exercise, or do both during the study. The study is taking place in Philadelphia, Pennsylvania.