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TIBSOVO is a prescribed medication approved to treat adult patients with myelodysplastic
syndromes (MDS) with an isocitrate dehydrogenase-1 (IDH1)
mutation
mutation:
Any change or alteration in a gene. A mutation may cause disease or may be a normal variation. Paroxysmal nocturnal hemoglobinuria (PNH) occurs because of a mutation in the PIG-A gene of a single stem cell in the bone…
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REBLOZYL® (luspatercept-aamt) was the first erythroid maturation agent (EMA) approved in
2020 by the Food and Drug Administration 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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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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Pure red cell aplasia (PRCA)
Pure red cell aplasia (PRCA):
A condition that occurs when the bone marrow stem cells do not make red blood cells. Red blood cell counts are low. White blood cell and platelet counts are normal.
is a condition in which the
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
stops…
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Summary of FDA-approved medicines for treating high risk MDS and transplantation options
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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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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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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)
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Explanation of how and why your MDS risk level determines your treatment options
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Supporting students with
Bone Marrow Failure
Bone Marrow Failure:
A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be…
Diseases, a guide for…
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Summary of active surveillance and
supportive care
supportive care:
Care given to improve the quality of life, or comfort, of a person with a chronic illness. Supportive care treats the symptoms rather than the underlying cause of a disease. The goal is to help the patient feel better. Patients with low blood counts may be given blood transfusions as supportive…
MDS treatment options
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SOLIRIS is a prescription medicine called 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 kinds of monocloncal antibodies. Each one looks for only one substance. Eculizumab (Soliris) is an monoclonal antibody that may be prescribed to treat patients with paroxysmal…
. SOLIRIS…
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Information about iron levels and the ways they fluctuate during treatment, alongside information about the effects of such fluctuations
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MarrowForums provides free discussion forums for patients with
bone marrow failure
bone marrow failure:
A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life…
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A list of organizations & programs that may provide financial resources & other assistance
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Summary for patients from the seventh International
Bone Marrow Failure
Bone Marrow Failure:
A condition that occurs when the bone marrow stops making enough healthy blood cells. The most common of these rare diseases are aplastic anemia, myelodysplastic syndromes (MDS) and paroxysmal nocturnal hemoglobinuria (PNH). Bone marrow failure can be acquired (begin any time in life) or can be…
Downloadable last updated
.
Learn more about how to find a
clinical trial
clinical trial:
A type of research study that tests how a drug, medical device, or treatment approach works in people. There are several types of clinical trials. Treatment trials test new treatment options. Diagnostic trials test new ways to diagnose a disease. Screening trials test the best way to detect a…
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