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Ravulizumab-cwvz
Ravulizumab-cwvz:
Ravulizumab-cwvz (Ultomiris®) is a drug approved by the U.S. Food and Drug Administration (FDA) in 2018 to treat PNH. ULTOMIRIS® is a long-acting C5 inhibitor that works by inhibiting the C5 protein in the terminal complement cascade. ULTOMIRIS® is a prescription medicine called a…
(Ultomiris®) is a drug approved by the U.S. Food…
Drug last updated
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Danicopan (ACH-4471)
Danicopan (ACH-4471):
Danicopan (ACH-4471) inhibits alternative pathway of complement (APC) activity, and has potential to block the alternative pathway of complement in paroxysmal nocturnal hemoglobinuria (PNH).
inhibits alternative pathway of complement (APC) activity, and has potential to block the alternative pathway of complement in
paroxysmal nocturnal…
Drug last updated
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Tamibarotene
Tamibarotene:
Tamibarotene (brand name: Amnolake), also called retinobenzoic acid, is orally active, synthetic retinoid, developed to overcome all-trans retinoic acid (ATRA) resistance, with potential antineoplastic activity against acute promyelocytic leukaemia (APL) .
(brand name: Amnolake), also called retinobenzoic acid, is orally active, synthetic retinoid, developed…
Drug last updated
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A macrolide compound obtained from Streptomyces hygroscopicus that acts by selectively blocking the transcriptional activation of cytokines thereby inhibiting cytokine production. It is bioactive only when bound to IMMUNOPHILINS.
Sirolimus
Sirolimus:
A macrolide compound obtained from Streptomyces hygroscopicus that acts by selectively blocking the transcriptional activation of…
Drug last updated
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APG-2575 is a novel, orally administered small-molecule Bcl-2‒selective inhibitor. APG-2575 is designed to treat hematologic malignancies and solid tumors by selectively blocking antiapoptotic protein Bcl-2 to restore the normal
apoptosis
apoptosis:
Programmed cell death.
process in cancer cells.
Drug last updated
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Alrizomadlin is an orally administered, selective, small-molecule inhibitor of the MDM2 protein. Alrizomadlin has strong binding affinity to MDM2 and is designed to activate tumor suppression activity of p53 by blocking the MDM2-p53 protein-protein interaction.
Drug last updated
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A methylated derivative and structural analog of PRIMA-1 (p53 re-activation and induction of massive
apoptosis
apoptosis:
Programmed cell death.
), with potential antineoplastic activity. Upon administration, PRIMA-1 analog
APR-246
APR-246:
A methylated derivative and structural analog of PRIMA-1 (p53 re-activation and induction of massive apoptosis), with potential…
Drug last updated
.
Hydroxyurea
Hydroxyurea:
Hydroxyurea is in a class of medications called antimetabolites. Hydroxyurea treats cancer by slowing or stopping the growth of cancer cells in your body. It can be used alone or with other medications to treat a certain types of leukemias.
is in a class of medications called antimetabolites. Hydroxyurea treats cancer by slowing or stopping the growth of…
Drug last updated
.
Iptacopan (LNP023)
Iptacopan (LNP023):
Iptacopan (LNP023) is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway. It is currently in clinical development for treating PNH.
is a first-in-class, orally administered, potent and highly selective factor B inhibitor of the alternative complement pathway. It is currently…
Drug last updated
.
Pozelimab
Pozelimab:
Pozelimab is a monoclonal antibody inhibiting the pathway associated with haemolysis of red blood cells. The drug is intended to decrease LDH levels and the incidence of breakthrough haemolysis.
is 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…
Drug last updated
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Tacrolimus
Tacrolimus:
Tacrolimus is in a class of medications called immunosuppressants. It works by decreasing the activity of the immune system. Tacrolimus can be prescribed to treat and prevent graft vs host disease (GVHD). It can also be used as part of combination therapy to treat aplastic anemia in place of…
is in a class of medications called immunosuppressants. It works by…
Drug last updated
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Folate
Folate:
(FOE-late) A B-vitamin that is found in fresh or lightly cooked green vegetables. It helps the bone marrow make normal blood cells. Most people get enough folate in their diet. Doctors may have people with paroxysmal nocturnal hemaglobinuria (PNH) take a man-made form of folate called folic acid.
is a B-vitamin that is found in fresh or lightly cooked green vegetables.…
Treatment last updated
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Clinical research
Clinical research:
A type of research that involves individual persons or a group of people. There are three types of clinical research. Patient-oriented research includes clinical trials which test how a drug, medical device, or treatment approach works in people. Epidemiology or behavioral studies look at the…
is at the heart of all medical advances, identifying new…
Treatment last updated
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Growth factors
Growth factors:
Growth factors are naturally occurring hormones in your body that signal your bone marrow to make more of certain types of blood cells. Man-made growth factors may be given to some people with bone marrow failure diseases to help increase red blood cell, white blood cell or platelet counts.
Red…
are naturally occurring hormones in your body that signal…
Treatment last updated
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Androgens
Androgens:
Androgens are natural male hormones that can cause your bone marrow to make more red blood cells. This can improve anemia. Androgens are sometimes used to treat aplastic anemia and PNH.
Androgens that may be used include:
Danazol (Danacrine®)
Fluoxymestrone (Halotestin®)
Oxymetholone
…
are natural male hormones that can cause your
bone marrow
bone…
Treatment last updated
.
Iron chelation therapy
Iron chelation therapy:
(kee-LAY-shun) A drug therapy to remove extra iron from the body. Patients with high blood iron (ferritin) levels may receive iron chelation therapy. The U.S. Food and Drug Administratin (FDA) has approved two iron chelators to treat iron overload in the U.S. - deferasirox, an oral iron chelator,…
is the main treatment used when you have a…
Treatment last updated
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If you have MDS and have a high-risk or intermediate-2 risk IPSS score, you have a higher risk of developing
Acute Myelogenous Leukemia
Acute Myelogenous Leukemia:
(uh-KYOOT my-uh-LAH-juh-nuss 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 numerous…
Treatment last updated
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