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Dr. Paibel Aguayo-Hiraldo, MD, anticipates your questions about inherited
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…
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Patient Ruth Cuadra describes her journey facing the challenges 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…
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Dr. Ryotaro Nakamura explains the entire process for
bone marrow
bone marrow:
The soft, spongy tissue inside most bones. Blood cells are formed in the bone marrow.
transplantation in this recorded session from the 2024 Los Angeles Patient and Family Conference. Learn the potential ways transplants can help patients with
aplastic anemia
aplastic anemia:
(ay-PLASS-tik uh-…
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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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In this webinar, Dr. Ronald Paquette discusses the latest advances in 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…
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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
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.…
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In this webinar, Dr. Akriti Jain will explains the latest research presented the American Society of Hematology meeting on MDS and secondary AML. This is an advanced presentation.
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We made it to another treatment day without anyone in our family getting sick! The week leading to treatment day has become pretty worrisome for me. What if someone in our family gets sick with or is exposed to COVID? Josh will not be able to get his infusion within the treatment window and then what? The questions I never thought I'd have to wonder about. I used to dread treatment day because it…
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I know my fiancé well. Even though he seemed to be healthy and his job kept him active, I knew it had been a long time since he had a medical physical, so I pushed him into an appointment.
Without symptoms, the doctor suggested routine blood work as a part of the physical. We were surprised that all his counts were low. The doctor decided to check again in a month, so I made…
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“Your blood counts are nearly nonexistent,” the doctor said.
Before this moment, Debra considered herself to be generally healthy, just tiring easily. She assumed the cause was the big project at work on top of a host of family and volunteer activities, or perhaps issues with her blood pressure. It was autumn of 2017.
She decided to checked out at the urgent care thinking they…
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2021 Treatment update
I am still taking the medication ultomiris, however, in late 2020 I was notified by Alexion (the drug company that makes ultomiris) that the concentration of ultomiris would be changing in 2021. By sometime in 2021 the original concentration of ultomiris will be phased out and will no longer available. The new concentration I would begin getting is 100mg/ml formulation. This…
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I was diagnosed with 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 low. Most…
on February 1, 2011, just 10…
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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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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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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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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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