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Here is Nick's story, as told by his mom, Pamela Karavite
At 13, Nick was an active teenaged boy. He played on a summer basketball team, and two baseball teams. I had seen bruises on his arms and legs, but they were typical for a teen boy that plays hard. Nick had just pitched his first no-hitter, and lethargically played in a couple of basketball games two days later. It…
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Deferiprone
Deferiprone:
Deferiprone is in a class of medications called iron chelators. It works by attaching to iron in the body so that it can be excreted (removed from the body).
Deferiprone comes in a pill form and is taken by mouth. Deferiprone is approved for use in Europe. Researchers in the U.S. are studying…
is in a class of medications called iron chelators. It works by…
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What is a Biosimilar?
A biosimilar is a type of biologic medication. The term “biosimilar” means that the medication is highly similar to the already approved brand name biologic (“originator” medication). Biosimilars are not generic versions of the original biologic.
Difference Between Generic Drugs and Biosimilars
Generic drugs have identical active ingredients to brand name drugs and are…
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Currently it is approved by the U.S. Food and Drug Administration (FDA) for use in treating a skin disease associated with leprosy.
Thalidomide
Thalidomide:
Currently it is approved by the U.S. Food and Drug Administration (FDA) for use in treating a skin disease associated with leprosy. Thalidomide is being studied, either as a single agent or in combination with other medicines, for…
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Venetoclax
Venetoclax:
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.
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…
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Sabatolimab
Sabatolimab:
Sabatolimab (MBG453) is a novel immuno-myeloid therapy that binds to TIM-3 on immune cells, facilitating antileukemic immune activation and phagocytic killing of leukemic cells. Sabatolimab also binds to TIM-3 on leukemic cells, potentially impeding self-renewal of LSCs via inhibition of TIM3…
(MBG453) is a novel immuno-myeloid therapy that binds to TIM-3 on…
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Mycophenolate mofetil
Mycophenolate mofetil:
Mycophenolate mofetil is approved by the U.S. Food and Drug Administration (FDA) to prevent organ rejection in people who have received a kidney, heart, or liver transplant. This medicine comes as a capsule, tablet, or liquid that is taken by mouth. Researchers are currently studying MMF in…
is approved by the U.S. Food and Drug…
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REBLOZYL® (luspatercept-aamt) is the first erythroid maturation agent (EMA) approved by the US Food and Drug Administration and European Union for the treatment of
anemia
anemia:
(uh-NEE-mee-uh) A condition in which there is a shortage of red blood cells in the bloodstream. This causes a low red blood cell count. Symptoms of anemia are fatigue and tiredness.
in adult patients with…
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A small molecule inhibitor of isocitrate dehydrogenase-1 (IDH1), which is mutated in several forms of cancer, including
acute myeloid leukemia
acute myeloid leukemia:
(uh-KYOOT my-uh-LOYD loo-KEE-mee-uh) A cancer of the blood cells. It happens when very young white blood cells (blasts) in the bone marrow fail to mature. The blast cells stay in the bone marrow and become to numerous.…
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Lenalidomide
Lenalidomide:
Lenalidomide is a capsule that is taken by mouth. It is approved for treating low-risk, transfusion-dependent myelodysplastic syndrome (MDS) patients with an abnormality of chromosome 5q. It is currently in clinical trials to test its efficacy with a broader range of MDS patients. For some MDS…
is a capsule that is taken by mouth. It is approved for treating…
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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.…
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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…
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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…
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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…
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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…
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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.
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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…
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