pediatric patient / parent

Doctors sometimes refer to particular drugs as an “active treatment” or “active drug therapy.” Active treatments treat the underlying cause of the

Doctors will sometimes refer to certain treatments as supportive therapy or supportive care. They help you manage the symptoms of your disease, although they do not treat the underlying cause of the disease.

These diseases can not be passed down through the genes from parent to child. Most cases of

How long do I have to live?

This is most people’s first question, along with “what can I expect to happen.” The doctor’s answer is called a prognosis (an educated guess about the likely course of your disease and how long you might live). Because each person is unique, and each person’s disease is different, a prognosis can be difficult to make. Also, how a disease progresses over time is unpredictable, and less is known about rare diseases than common diseases.

PNH is considered a chronic disease meaning that it lasts a long time. The only potential cure is a

MDS treatment is designed to increase the number of healthy cells in your blood (blood count). When your blood counts go up you are less likely to need blood from a donor (transfusion), your quality of life becomes better and your symptoms are not as bad.

AML (

PNH can strike people of any age, race or gender. Only about 500 people are diagnosed with PNH in the U.S. each year. PNH tends to be more common in adults in their thirties and early forties.

PNH (