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Descriptions of the criteria to determine severity of
aplastic anemia
aplastic anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most…
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As you search online, you will find websites from many different sources. These seven questions will help you analyze and evaluate the credibility of each site you review. Many of these details can also be found in a website's "About Us" or "Contact Us" section.
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What is EMPAVELI®? EMPAVELI® (previously known as
pegcetacoplan
pegcetacoplan:
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 skin (subcutaneously) by using the Empaveli injector or with an…
) is the…
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Chronic lymphocytic leukemia (CLL)
Chronic lymphocytic leukemia (CLL):
(CRAW-nik lim-foe-SIGH-tick loo-KEE-mee-uh) Chronic lymphocytic leukemia is a type of blood, lymph node and bone marrow cancer in which too many abnormal white blood cells called lymphocytes accumulate in your body.
is a type of cancer that involves certain white blood cells (lymphocytes) that develop in the
bone…
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Would you like to connect with other patients and families in your own town? Learn how you can participate in our volunteer-led Community Connections-local groups designed to inspire the creation of community organizations for the purpose of connecting and supporting patients and families, as well as raising awareness and funds for AAMDSIF programs.
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Form to keep track of
blood transfusions
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…
as part of
aplastic anemia…
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Form to keep track of
blood transfusions
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…
as part of PNH treatment
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A patient's guide to understanding blood and
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
. Blood is a "circulating organ" of the body. It is composed of many different types of cells that are carried through the body in
plasma
plasma:
The fluid part of the blood. Plasma is mostly made of water with…
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This essential primer for patients and families on
aplastic anemia
aplastic anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most……
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Summary of FDA-approved medicines for treating
aplastic anemia
aplastic anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most…
and…
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Learn more about the connection between
aplastic anemia
aplastic anemia:
(ay-PLASS-tik uh-NEE_mee-uh) A rare and serious condition in which the bone marrow fails to make enough blood cells - red blood cells, white blood cells, and platelets. The term aplastic is a Greek word meaning not to form. Anemia is a condition that happens when red blood cell count is low. Most…
and other…
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Summary of FDA-approved medicines for treating PNH and transplantation options.
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Form to record symptoms or reactions to treatment and medications that can be shared with health care providers
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This guide provides information about
clinical trials
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.
Understanding Clinical Trials
Clinical…
and gives you some things…
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Guidelines for managing your MDS treatment plan with your health care team
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Information on workplace rights & responsibilities for individuals 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. Hemoglobinuria means hemoglobin in…
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RYTELO (imetelstat) is an oligonucleotide telomerase inhibitor used to treat adults with IPSS-R low to intermediate-1
myelodysplastic syndromes
myelodysplastic syndromes:
(my-eh-lo-diss-PLASS-tik SIN-dromez) A group of disorders where the bone marrow does not work well, and the bone marrow cells fail to make enough healthy blood cells. Myelo refers to the bone marrow. Dysplastic means…
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This guide provides important information about
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
and stem cell transplantation
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