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Introductory image: March for Marrow - Team Krissie




Androgens are natural male hormones that can cause your bone marrow to make more red blood cells. This can improve anemia. Androgens are sometimes used to treat aplastic anemia and PNH.

Blood Transfusions

A blood transfusion is a safe and common procedure. Most people who have a bone marrow failure disease like aplastic anemia, MDS or PNH will receive at least one blood transfusion. When you receive a blood transfusion, parts of blood from a donor are put into your bloodstream. This can help some patients with low blood counts.


Eculizumab (Soliris ®) was the first drug approved by the U.S. Food and Drug Administration (FDA) and the European Medicines Evaluation Agency (EMEA) to treat PNH. It works by making your complement system less active and reduces hemolysis Soliris® is approved for the treatment of patients with PNH in nearly 50 countries worldwide.

Folic Acid

Folic acid, also called folate or vitamin B-12, is found in fresh or lightly cooked green vegetables. It helps your bone marrow make normal blood cells. When your bone marrow has to make more cells, it needs a larger supply of folic acid.

Most people get enough folate in their diet. But if you have PNH, it's a good idea to take 1 mg each day of a man-made form of folate called folic acid.

Growth Factors

Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts.

Iron Chelation

Iron chelation therapy is the main treatment used when you have a condition called iron overload. Iron overload means you have too much iron in your body. This can be a problem for people who get lots of red blood cell transfusions.

Pegcetacoplan (EMPAVELI®)

EMPAVELI is the first PNH treatment that binds to complement protein C3. It was approved by the Food and Drug Administration in May 2021 for treating adult patients with paroxysmal nocturnal hemoglobinuria (PNH). EMPAVELI is given by infusion under the skin (subcutaneously) into your stomach (abdomen), back of upper arms, hips, or thighs using an infusion pump.

Ravulizumab-cwvz (Ultomiris®)

Ravulizumab-cwvz (Ultomiris®) is a drug approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat PNH. ULTOMIRIS® is a long-acting C5 inhibitor that works by inhibiting the C5 protein in the terminal complement cascade. On June 7, 2021, the U.S. FDA approved the expanded use of ULTOMIRIS® to include children (one month of age and older) and adolescents with PNH.  


Bone Marrow Transplant

A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure. But for many patients, a BMT is not an option due to the risks and potential long-term side effects as an "imperfect cure".

Clinical Trials

Clinical research is at the heart of all medical advances, identifying new ways to prevent, detect or treat disease. If you have a bone marrow failure disease, you may want to consider taking part in a clinical trial, also called a research study.

Have you been wondering how to get involved with PNH research but did not know where to start? Here’s your chance! Check out the Global PNH Patient Registry at:
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