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PNH occurs because of a genetic change in
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
stem cells
stem cells:
Cells in the body that develop into other cells. There are two main sources of stem cells. Embryonic stem cells come from human embryos and are used in medical research. Adult stem cells in the body…
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PNH can cause variety of symptoms. These can vary greatly from person to person. You may have many symptoms, or just one or two. And you may get a new symptom at any point in the course of your illness. The severity and number of symptoms are often related to the size of your PNH
clone
clone:
To make copies. Bone marrow stem cells clone themselves all the time. The cloned stem cells…
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AAMDSIF provides educational materials for patients and healthcare professionals written in plain language and reviewed by highly respected and recognized medical doctors who are experts in
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,…
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In this webinar, Dr. Firas El Chaer discusses the diagnosis of secondary AML and how treatment options are decided. Secondary
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…
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Dr. Daria Babushok discusses the significance of PNH clones in the diagnostic assessment of 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…
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Tiffany Tanaka, MD (UC San Diego Health) helps MDS patients understand the most common mutations and how genetic testing can help patients get the best possible treatment.
Session recorded as part of the Spring 2021 Virtual Patient & Family Conference.
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In this webinar, Dr. David Beck, MD, PhD and Anna Cantor, MA, MS, CGC, discuss VEXAS syndrome which is a newly discovered auto-inflammatory condition affecting older adults. VEXAS syndrome often overlaps with
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…
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In this webinar, Dr. Phillip Scheinberg discusses research most relevant to patients reported over the past year on the biology and treatment 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…
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Dr. Catherine Lee will provide a comprehensive overview of chronic and acute graft vs host disease ("GvHD") which is a complication of
bone marrow transplant
bone marrow transplant:
A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers…
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Dr. Naval Daver describes recent research and upcoming treatments for MDS presented at the ASCO and EHA 2022 meetings. Discover how the understanding of MDS, or
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…
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Dr. Patel explains the challenges and successes 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.…
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Dr. Mikhail Kogan, Medical Director of the GW Center for Integrative Medicine, discusses the differences between integrative, holistic and functional medicines and how they can be used to treat patients. The end of the webinar features questions from patients in the audience.
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In this two-part conference session, Dr. Brion Randolph discusses the process of transplant as a modality 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…
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In this conference session, Dr. Sharma discusses what causes severe
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…
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In this discussion, Dr. Nandita Khera of Associate Professor of Mayo Clinic and Dr. Linda Burns Sr. Scientific Director of CIBMTR discuss which treatments are available to older MDS patients.
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In this webinar Drs. Linda Burns and Nandita Khera will discuss the advances in transplantation for older patients with MDS. Stem cell transplants are not frequently offered to older patients with high-risk
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…
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Dr. Jorg Dietrich will help patients and their loved ones understand the chemistry and biology behind what happens to patient brains when
chemotherapy
chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People with high-risk or intermediate-2 risk myelodysplastic syndrome (MDS) may be given chemotherapy to kill bone marrow cells that have an…
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Has a
bone marrow transplant
bone marrow transplant:
A bone marrow transplant (BMT) is also called a stem cell transplant (SCT) or hematopoietic stem cell transplant (HSCT).The procedure replaces unhealthy blood-forming stem cells with healthy ones and offers some patients the possibility of a cure. But for many patients, a BMT is not an option due…
been suggested as a treatment for…
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In this conference session, Dr. Wlodarski discusses the basics of Pediatric MDS, the differences between MDS in children and adults, and practical examples on GATA2 and SAMD9/9L.
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