The National Heart, Lung, and Blood Institute (NHLBI) is strongly committed to supporting research aimed at preventing and treating heart, lung, and blood diseases and conditions and sleep disorders.
NHLBI-supported research has led to many advances in medical knowledge and care. For example, this research has uncovered some of the causes of blood disorders, as well as ways to prevent and treat these disorders.
Many questions remain about blood disorders, including thalassemias. The NHLBI continues to support research to learn more. For example, NHLBI-sponsored research includes studies that explore:
- Ways to make gene therapy possible for treating people who have thalassemias
- How to improve blood and marrow stem cell transplants for people who have thalassemias and other severe forms of anemia
Much of the NHLBI’s research depends on the willingness of volunteers to take part in clinical trials. Clinical trials test new ways to prevent, diagnose, or treat various diseases and conditions.
For example, new treatments for a disease or condition (such as medicines, medical devices, surgeries, or procedures) are tested in volunteers who have the illness. Testing shows whether a treatment is safe and effective in humans before it is made available for widespread use.
By taking part in a clinical trial, you can gain access to new treatments before they're widely available. You also will have the support of a team of health care providers, who will likely monitor your health closely. Even if you don't directly benefit from the results of a clinical trial, the information gathered can help others and add to scientific knowledge.
If you volunteer for a clinical trial, the research will be explained to you in detail. You'll learn about treatments and tests you may receive, and the benefits and risks they may pose. You'll also be given a chance to ask questions about the research. This process is called informed consent.
If you agree to take part in the trial, you'll be asked to sign an informed consent form. This form is not a contract. You have the right to withdraw from a study at any time, for any reason. Also, you have the right to learn about new risks or findings that emerge during the trial.
For more information about clinical trials related to thalassemias, talk with your doctor. You also can visit the following Web sites to learn more about clinical research and to search for clinical trials:
For more information about clinical trials for children, visit the NHLBI's Children and Clinical Studies Web page.
Visit Children and Clinical Studies to hear experts, parents, and children talk about their experiences with clinical research.