Aplastic Anemia

Clinical Trial: A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a disease or health problem. Quality of life (supportive care) trials study ways to improve the comfort of people with chronic illness. Prevention trials look for better ways to prevent disease in people who have never had the disease. Trials are in four phases. Phase I tests a new drug or treatment in a small group to see if it is safe. Phase II expands the study to a larger group of people to find out if it works. Phase III expands the study to an even larger group of people to compare it to the standard treatment for the disease; and Phase IV takes place after the drug or treatment has been licensed and marketed to find out the long-term impact of the new treatment. Low Dose Danazol for the Treatment of Telomere Related Diseases

Status
Recruiting
Study Date (Range)
-
Bone Marrow Disease(s)
Aplastic Anemia
Age Group
3 years and older
DNA is a structure in the body. It contains data about how the body develops and works. Telomeres are found on the end of chromosomes in DNA. Some people with short telomeres or other gene changes can develop diseases of the bone marrow, lung, and liver. Researchers at the…

As Patient Advocate, Emma Makes New Friends

When Emma was still being seen at the NIH twice a week, we were talking with her physician’s assistant about her t-shirt fundraiser and her Facebook group. After hearing what Emma was doing with these activities, they asked her to be their new pediatric patient advocate, helping newer pediatric patients with advice on how to cope with treatments. This seemed to be another way we could help.

My Encounter with Aplastic Anemia

In 2007, when I was 25 years old, I started to get winded going up a flight of stairs. I also began having two hour long nosebleeds, and I knew something was wrong. I had my bloodwork checked and shortly after my doctor told me to go to the hospital right away because my blood counts were extremely low. I had no idea what that meant at the time, and wasn’t overly alarmed.

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