As someone living with aplastic anemia, myelodysplastic syndrome, or paroxysmal nocturnal hemoglobinuria, you may be interested in participating in a clinical trial. Clinical trials can be a valuable option, but they are not for everyone. Before joining a trial, you should understand all the potential risks and the possible benefits.
Clinical trials are research studies that look at how well a medication or a procedure will work in patients in the hope of finding a better way to treat diseases. Clinical trials may also compare how one treatment works in relation to another treatment.
Clinical trials are particularly important for rare diseases like aplastic anemia, MDS, and PNH and typically need many patients. (Different trials need different patients who have had different types of treatments; the study description will tell you what patients are eligible for the trial.) Behind every successful treatment option for a disease is a clinical trial—and the subjects who participated in that study. Trials have led to the approval of a number of effective drugs (like Dacogen®, Exjade®, Revlimid®, Soliris®, and Vidaza®) and procedures (like bone marrow transplants), but not all patients do well with the available treatment options. Clinical trials, while open to a number of people, can be of particular interest to such patients and may hold the key to success for them.
Clinical Trials Explained
Finding a Clinical Trial
Patient Travel Fund
Research Consortium
For Researchers
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