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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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PNH patients need to take a few special precautions
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VOYDEYA is a prescription medicine used along with ravulizumab or
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…
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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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Basic description of PNH and how it affects your
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
and blood
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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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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 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…
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Basic description of MDS and how it affects your
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
and blood
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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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Form to keep track of
platelet
platelet:
The smallest type of blood cell. Platelets help the blood to clot and stop bleeding. Also called a thrombocyte.
transfusions as part of PNH treatment
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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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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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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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Summary of the common side effects of MDS treatments and tips for managing them
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Review of the range of MDS symptoms based on low blood counts
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INQOVI® (previously known as ASTX727) is a prescribed oral tablet combination of
decitabine
decitabine:
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 is…
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A list of organizations & programs that may provide financial resources & other assistance
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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…
occurs when blood-forming
stem cells
stem cells…
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