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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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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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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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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
.
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
.
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
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Cytarabine (ara-C)
Cytarabine (ara-C):
Cytarabine (ara-C) is a chemotherapy drug. This medicine reduces the growth of cancer cells and can suppress the immune system. It is used for treating leukemias or lymphomas. It is often given with other cancer drugs.
is a
chemotherapy
chemotherapy:
(kee-moe-THER-uh-pee) The use of medicines that kill cells (cytotoxic agents). People…
Drug last updated
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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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It is used to prevent blood clots from forming.
Warfarin
Warfarin:
It is used to prevent blood clots from forming. Warfarin is a tablet that is taken by mouth.
is a tablet that is taken by mouth.
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
.
Prednisone
Prednisone:
Prednisone a man-made version of a hormone produced naturally by the adrenal gland that can help reduce inflammation and stop allergic responses. It is prescribed when the body is not producing enough of this chemical on its own.
How does it work?
For PNH, it may be used to decrease…
a man-made version of a
hormone
hormone:
A part of the endocrine…
Drug last updated
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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
.
Heparin
Heparin:
Heparin is in a class of medications called anticoagulants ('blood thinners'). It works by decreasing the clotting ability of the blood. Heparin is also used to stop the growth of clots that have already formed in the blood vessels, but it cannot be used to decrease the size of clots that have…
is in a class of medications called anticoagulants ('blood thinners'). It…
Drug last updated
.
Enoxaparin
Enoxaparin:
Enoxaparin is in a class of medications called low molecular weight heparins. It works by stopping the formation of substances that cause clots. It is also used in combination with warfarin to treat blood clots in the leg.
is in a class of medications called low molecular weight heparins. It works by stopping the formation of substances that cause…
Drug last updated
.
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
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