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More drugs are available to treat bone marrow failure disease than ever before. Researchers constantly look for ways to enhance drugs already on the market and develop new ones with fewer side effects. Some drugs treat only one type of the disease, while others may be used to treat more than one type. Check with your doctor to find out which drugs are right for you and why.


Brand Name: REBLOZYL®
Treatment for: myelodysplastic syndromes (MDS), myeloproliferative neoplasms (MPN)
First erythroid maturation agent (EMA) approved by the FDA for the treatment of anemia in adult patients with myelodysplastic syndromes (MDS) who have been failed an erythropoiesis stimulating agent (ESA) or are unlikely to respond to an ESA, and are requiring transfusions of 2 or more red blood cell (RBC) units over 8 weeks.

Mycophenolate Mofetil

Brand Name: CellCept
Treatment for: aplastic anemia
A drug that lowers the body's immune response, also referred to as an immunosuppressant drug.


Brand Name: EMPAVELI
Treatment for: paroxysmal nocturnal hemoglobinuria (PNH)
EMPAVELI is the first PNH treatment that binds to compliment 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.


Brand Name: Prograf
Treatment for: aplastic anemia


Brand Name: Thalomid
Treatment for: myelodysplastic syndromes (MDS)
Thalidomide is an old drug, though it has not been widely available since the 1960s, when it was found to cause birth defects.


Brand Name: Venclexta
Treatment for: chronic lymphocytic leukemia (CLL), myelodysplastic syndromes (MDS)
Venetoclax is used to treat chronic lymphocytic leukemia or small lymphocytic leukemia in adults. Venetoclax is used alone or in combination with other cancer medicines to treat these conditions.


Brand Name: Coumadin
Treatment for:
A blood thinner, or anticoagulant.

AAMDSIF does not recommend, endorse, or make any representation about the efficacy, appropriateness or suitability of any drug, treatment or therapy listed on this website. Some therapies listed on our site are considered experimental for the treatment of bone marrow failure diseases. Always seek the advice of your physician or other qualified health care provider with any questions you may have regarding any therapy, and never disregard professional medical advice or delay in seeking it because of something you have read on this website.

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