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Iptacopan
Iptacopan, sold under the brand name Fabhalta, is a medication used for the treatment of paroxysmal nocturnal hemoglobinuria (PNH). It is a complement factor B inhibitor that was developed by Novartis. It is taken by mouth. Iptacopan was approved by the US Food and Drug Administration (FDA) for the treatment of adults with paroxysmal nocturnal hemoglobinuria in December 2023. The FDA considers it to be a first-in-class medication. Medical uses Iptacopan is indicated for the treatment of adults with paroxysmal nocturnal hemoglobinuria. Mechanism of action Iptacopan binds to Factor B of the alternative complement pathway and regulates the cleavage of C3, generation of downstream effectors, and the amplification of the terminal pathway. In PNH, intravascular hemolysis (IVH) is mediated by the downstream membrane attack complex (MAC), while extravascular hemolysis (EVH) is facilitated by C3b opsonization. Iptacopan acts proximally in the alternative pathway of the c ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease of the blood characterized by destruction of red blood cells by the complement system, a part of the body's innate immune system. This destructive process occurs due to deficiency of the red blood cell surface protein DAF, which normally inhibits such immune reactions. Since the complement cascade attacks the red blood cells within the blood vessels of the circulatory system, the red blood cell destruction (hemolysis) is considered an ''intravascular'' hemolytic anemia. Other key features of the disease, such as the high incidence of venous blood clot formation, are incompletely understood. PNH is the only hemolytic anemia caused by an ''acquired'' (rather than inherited) intrinsic defect in the cell membrane (deficiency of glycophosphatidylinositol or GPI) leading to the absence of protective exterior surface proteins that normally attach via a GPI anchor. It may develop on its own ("primary ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Paroxysmal Nocturnal Hemoglobinuria
Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, acquired, life-threatening disease of the blood characterized by destruction of red blood cells by the complement system, a part of the body's innate immune system. This destructive process occurs due to deficiency of the red blood cell surface protein DAF, which normally inhibits such immune reactions. Since the complement cascade attacks the red blood cells within the blood vessels of the circulatory system, the red blood cell destruction (hemolysis) is considered an ''intravascular'' hemolytic anemia. Other key features of the disease, such as the high incidence of venous blood clot formation, are incompletely understood. PNH is the only hemolytic anemia caused by an ''acquired'' (rather than inherited) intrinsic defect in the cell membrane (deficiency of glycophosphatidylinositol or GPI) leading to the absence of protective exterior surface proteins that normally attach via a GPI anchor. It may develop on its own ("primary ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
By Mouth
Oral administration is a route of administration where a substance is taken through the mouth. Per os abbreviated to P.O. is sometimes used as a direction for medication to be taken orally. Many medications are taken orally because they are intended to have a systemic effect, reaching different parts of the body via the bloodstream, for example. Oral administration can be easier and less painful than other routes, such as injection. However, the onset of action is relatively low, and the effectiveness is reduced if it is not absorbed properly in the digestive system, or if it is broken down by digestive enzymes before it can reach the bloodstream. Some medications may cause gastrointestinal side effects, such as nausea or vomiting, when taken orally. Oral administration can also only be applied to conscious patients, and patients willing and able to swallow. Terminology ''Per os'' (; ''P.O.'') is an adverbial phrase meaning literally from Latin "through the mouth" or "by mou ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Haemophilus Influenzae
''Haemophilus influenzae'' (formerly called Pfeiffer's bacillus or ''Bacillus influenzae'') is a Gram-negative, non-motile, coccobacillary, facultatively anaerobic, capnophilic pathogenic bacterium of the family Pasteurellaceae. The bacteria are mesophilic and grow best at temperatures between 35 and 37℃. ''H. influenzae'' was first described in 1892 by Richard Pfeiffer during an influenza pandemic when he incorrectly described ''Haemophilus influenzae'' as the causative microbe, which is why the bacteria retain the name "influenza". ''H. influenzae'' is responsible for a wide range of localized and invasive infections, typically in infants and children, including pneumonia, meningitis, or bloodstream infections. Treatment consists of antibiotics, however ''H. influenzae'' is often resistant to the penicillin family but augmentin can be used in mild cases. The recommended form of prevention is a series of the Hib vaccine and boosters, which are most often given under the ag ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Benzoic Acids
Benzoic acid is a white (or colorless) solid organic compound with the formula , whose structure consists of a benzene ring () with a carboxyl () substituent. It is the simplest aromatic carboxylic acid. The name is derived from gum benzoin, which was for a long time its only source. Benzoic acid occurs naturally in many plants and serves as an intermediate in the biosynthesis of many secondary metabolites. Salts of benzoic acid are used as food preservatives. Benzoic acid is an important precursor for the industrial synthesis of many other organic substances. The salts and esters of benzoic acid are known as benzoates . History Benzoic acid was discovered in the sixteenth century. The dry distillation of gum benzoin was first described by Nostradamus (1556), and then by Alexius Pedemontanus (1560) and Blaise de Vigenère (1596). Justus von Liebig and Friedrich Wöhler determined the composition of benzoic acid. These latter also investigated how hippuric acid is related ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Methoxy Compounds
In organic chemistry, a methoxy group is the functional group consisting of a methyl group bound to oxygen. This alkoxy group has the formula . On a benzene ring, the Hammett equation classifies a methoxy substituent at the ''para'' position as an electron-donating group, but as an electron-withdrawing group if at the ''meta'' position. At the ''ortho'' position, steric effects are likely to cause a significant alteration in the Hammett equation prediction which otherwise follows the same trend as that of the ''para'' position. Occurrence The simplest of methoxy compounds are methanol and dimethyl ether. Other methoxy ethers include anisole and vanillin. Many alkoxides contain methoxy groups, e.g. tetramethyl orthosilicate and titanium methoxide. Such compounds are often classified as methoxides. Esters with a methoxy group can be referred to as methyl esters, and the —COOCH3 substituent is called a methoxycarbonyl. Biosynthesis In nature, methoxy groups are found on nucleosi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Piperidines
Piperidine is an organic compound with the molecular formula (CH2)5NH. This heterocyclic amine consists of a six-membered ring containing five methylene bridges (–CH2–) and one amine bridge (–NH–). It is a colorless liquid with an odor described as objectionable, and typical of amines. The name comes from the genus name ''Piper'', which is the Latin word for pepper. Although piperidine is a common organic compound, it is best known as a representative structure element within many pharmaceuticals and alkaloids, such as natural-occurring solenopsins. Production Piperidine was first reported in 1850 by the Scottish chemist Thomas Anderson and again, independently, in 1852 by the French chemist Auguste Cahours, who named it. Both of them obtained piperidine by reacting piperine with nitric acid. Industrially, piperidine is produced by the hydrogenation of pyridine, usually over a molybdenum disulfide catalyst: : C5H5N + 3 H2 → C5H10NH Pyridine can also be reduce ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Indoles
Indole is an aromatic heterocyclic organic compound with the formula C8 H7 N. It has a bicyclic structure, consisting of a six-membered benzene ring fused to a five-membered pyrrole ring. Indole is widely distributed in the natural environment and can be produced by a variety of bacteria. As an intercellular signal molecule, indole regulates various aspects of bacterial physiology, including spore formation, plasmid stability, resistance to drugs, biofilm formation, and virulence. The amino acid tryptophan is an indole derivative and the precursor of the neurotransmitter serotonin. General properties and occurrence Indole is a solid at room temperature. It occurs naturally in human feces and has an intense fecal odor. At very low concentrations, however, it has a flowery smell, and is a constituent of many perfumes. It also occurs in coal tar. The corresponding substituent is called indolyl. Indole undergoes electrophilic substitution, mainly at position 3 (see diagram i ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Immunosuppressants
Immunosuppressive drugs, also known as immunosuppressive agents, immunosuppressants and antirejection medications, are drugs that inhibit or prevent activity of the immune system. Classification Immunosuppressive drugs can be classified into five groups: * glucocorticoids * cytostatics * antibodies * drugs acting on immunophilins * other drugs Glucocorticoids In pharmacologic (supraphysiologic) doses, glucocorticoids, such as prednisone, dexamethasone, and hydrocortisone are used to suppress various allergic, inflammatory, and autoimmune disorders. They are also administered as posttransplantory immunosuppressants to prevent the acute transplant rejection and graft-versus-host disease. Nevertheless, they do not prevent an infection and also inhibit later reparative processes. Immunosuppressive mechanism Glucocorticoids suppress cell-mediated immunity. They act by inhibiting genes that code for the cytokines Interleukin 1 (IL-1), IL-2, IL-3, IL-4, IL-5, IL-6, IL-8 ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Hematology
Hematology ( always spelled haematology in British English) is the branch of medicine concerned with the study of the cause, prognosis, treatment, and prevention of diseases related to blood. It involves treating diseases that affect the production of blood and its components, such as blood cells, hemoglobin, blood proteins, bone marrow, platelets, blood vessels, spleen, and the mechanism of coagulation. Such diseases might include hemophilia, blood clots (thrombus), other bleeding disorders, and blood cancers such as leukemia, multiple myeloma, and lymphoma. The laboratory analysis of blood is frequently performed by a medical technologist or medical laboratory scientist. Specialization Physicians specialized in hematology are known as hematologists or haematologists. Their routine work mainly includes the care and treatment of patients with hematological diseases, although some may also work at the hematology laboratory viewing blood films and bone marrow slides under the ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Glomerulopathy
Glomerulopathy is a set of diseases affecting the glomeruli of the nephron. Such diseases can include processes that are inflammatory or noninflammatory. Because the term ''glomerulitis'' exists for inflammatory conditions, ''glomerulopathy'' sometimes carries a noninflammatory implication. References External links Kidney diseases {{genitourinary-disease-stub ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
