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The U.S. Food and Drug Administration (FDA) approved
fludarabine
fludarabine:
The U.S. Food and Drug Administration (FDA) approved fludarabine for treating chronic lymphocytic leukemia (CCL). Researchers are studying fludarabine in combination with other medicines for treating bone marrow failure.
for treating chronic lymphocytic leukemia (CCL). Researchers are studying fludarabine in…
Drug last updated
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FABHALTA, a complement factor B inhibitor, is the first oral medication approved to treat adults with
paroxysmal nocturnal hemoglobinuria
paroxysmal nocturnal hemoglobinuria:
(par-uk-SIZ-muhl nok-TURN-uhl hee-muh-gloe-buh-NYOOR-ee-uh) A rare and serious blood disease that causes red blood cells to break apart. Paroxysmal means sudden and irregular. Nocturnal means at night.…
Drug last updated
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It is known as a hematopoietic
growth factor
growth factor:
A substance made by the body that stimulates the bone marrow to produce blood cells. Some growth factors are man-made in the laboratory and used for treating low blood counts. These include red blood cell growth factors called erythropoietin (EPO) and darbepoetin, and white blood cell growth…
or colony stimulating factorand is…
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Epoetin alfa
Epoetin alfa:
Epoetin alfa can help improve red blood cell counts in bone marrow failure disease patients whose natural erythropoietin levels are low. It is given by injection under the skin (subcutaneous) or in the vein (intravenous). Epoetin alfa is approved by the U.S. Food and Drug Administration (FDA) for…
can help improve
red blood cell
red blood cell:
The…
Drug last updated
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What are the possible side effects of eltrombopag (Promacta)?
Get emergency medical help if you have any of these signs of an allergic reaction: hives; difficult breathing; swelling of your face, lips, tongue, or throat.
Stop using
eltrombopag
eltrombopag:
What are the possible side effects of eltrombopag (Promacta)?
Get emergency medical help if you have any of these signs of an…
Drug last updated
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Deferoxamine
Deferoxamine:
Deferoxamine is an iron-binding agent that belongs to a class of drugs known as heavy metal antagonists. It works by helping the kidneys and gallbladder get rid of the extra iron.
is an iron-binding agent that belongs to a class of drugs known as heavy metal antagonists. It works by helping the kidneys and gallbladder get rid of the extra iron.
Drug last updated
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Eculizumab
Eculizumab:
Eculizumab (Soliris ®) is given as an IV into a vein at the doctor’s office or at a special center. The procedure usually takes about 35 minutes. You will probably get an IV once a week for the first 4 weeks. Starting in the 5th week, you will get a slightly higher dose of Soliris every 2 weeks.
…
(Soliris ®) is given as an IV into a vein at the doctor’s office…
Drug last updated
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It comes in a tablet form which is dissolved in juice or water and taken by mouth. Iron build-up can occur in patients who get a lot of
red blood cell
red blood cell:
The most numerous type of blood cell in healthy people. Red blood cells contain hemoglobin, a protein that picks up oxygen in the lungs and brings it to cells in all parts of the body. Also called erythrocyte, RBC.…
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INQOVI® for adult patients with MDS or CMML who are intermediate-1, intermediate-2, and high-risk IPSS groups. It is a prescribed oral tablet combination of decitabine and cedazurine approved by the FDA for adult patients with myelodysplastic syndromes (MDS) and patients with chronic myelomonocytic leukemia (CMML) including:
previously treated and…
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It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely.
Decitabine
Decitabine:
It works by reducing the amount of methylation in the body. Methylation is a process that acts like…
Drug last updated
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It is in a class of medicines called erythropoiesis-stimulating agents (ESAs).
Darbepoetin alfa
Darbepoetin alfa:
It is in a class of medicines called erythropoiesis-stimulating agents (ESAs). Darbepoetin alfa can help improve red blood cell counts in bone marrow failure patients whose natural erythropoietin levels are low. It is given by injection under the skin (subcutaneous) or in…
Drug last updated
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Cyclosporine
Cyclosporine:
Cyclosporine is used along with antithymocyte globulin (ATG), another immunosuppressant, for treating aplastic anemia and some other forms of bone marrow failure.
is used along with
antithymocyte globulin
antithymocyte globulin:
ATG is an immunosuppressant, a drug that lowers the body's immune response. It is typically used with cyclosporine as the…
Drug last updated
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It works by reducing the amount of methylation in the body. Methylation is a process that acts like a switch to turn off or “silence” genes in certain cells. When these genes (called tumor suppressor genes) are turned off, MDS cells and cancer cells can grow freely.
Azacitidine
Azacitidine:
It works by reducing the amount of methylation in the body. Methylation is a process that acts…
Drug last updated
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Cyclophosphamide
Cyclophosphamide:
Cyclophosphamide is in a class of medications called alkylating agents. When used to treat cancer, it works by slowing or stopping the growth of cancer cells in your body. When cyclophosphamide is used to treat bone marrow failure, it works by suppressing your body's immune system.
is in a class of medications called alkylating agents. When used to…
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Romiplostim
Romiplostim:
Romiplostim is in a class of medications called thrombopoietin receptor agonists. It works by causing the cells in the bone marrow to produce more platelets. Romiplostim injection is used to increase the number of platelets enough to lower the risk of bleeding, but it is not used to increase the…
is in a class of medications called thrombopoietin receptor…
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Daunorubicin
Daunorubicin:
Daunorubicin is in a class of medications called anthracyclines. It works by slowing or stopping the growth of cancer cells in your body. It is used in combination with other chemotherapy drugs to treat a certain type of acute myeloid leukemia (AML).
is in a class of medications called anthracyclines. It works by slowing or stopping the growth…
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Cemdisiran (ALN-CC5)
Cemdisiran (ALN-CC5):
Cemdisiran (ALN-CC5) is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway in development for the treatment of complement-mediated diseases.
is a subcutaneously administered, investigational RNAi therapeutic targeting the C5 component of the complement pathway in development for…
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Pozelimab
Pozelimab:
Pozelimab is a monoclonal antibody inhibiting the pathway associated with haemolysis of red blood cells. The drug is intended to decrease LDH levels and the incidence of breakthrough haemolysis.
is a
monoclonal antibody
monoclonal antibody:
A type of protein called an antibody that is engineered to look for a specific substance in the body. There are many…
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Iptacopan (LNP023)
Iptacopan (LNP023):
Iptacopan (LNP023) is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway. It is currently in clinical development for treating PNH.
is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway. It is currently…
Drug last updated
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