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Glycerol Phenylbutyrate
Glycerol phenylbutyrate (USAN), trade name Ravicti, is a medication used in the treatment of certain inborn urea cycle disorders. The medication works by preventing the harmful buildup of ammonia in the body. It is an FDA-approved prescription drug in the US. It is approved for anyone over 2 months of age. It was developed by Hyperion Therapeutics based on the existing drug Buphenyl, and received approval on February 1, 2013. Hyperion has been criticized for setting a high price for the drug. The price was set at US$250,000–290,000. In 2014, the drug generated $30.8 million in net sales, far behind the older and less expensive Buphenyl ($113.6 million in sales). In March 2015, Horizon Pharma Horizon Therapeutics Public Ltd Co is a biopharmaceutical company focused on researching, developing, and commercializing medicines that address critical needs for people impacted by rare and rheumatic diseases. Horizon primarily markets products ... acquired Hyperion Therapeutics and th ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Oral Administration
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 mouth ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
United States Adopted Name
A United States Adopted Name (USAN) is a unique nonproprietary name assigned to a medication marketed in the United States. Each name is assigned by the USAN Council, which is co-sponsored by the American Medical Association (AMA), the United States Pharmacopeial Convention (USP), and the American Pharmacists Association (APhA). The USAN Program states that its goal is to select simple, informative, and unique nonproprietary names (also called generic names) for drugs by establishing logical nomenclature classifications based on pharmacological or chemical relationships. In addition to drugs, the USAN Council names agents for gene therapy and cell therapy, List of soft contact lens materials, contact lens polymers, surgical materials, diagnostics, carriers, and substances used as an excipient. The USAN Council works in conjunction with the World Health Organization (WHO) international nonproprietary name (INN) Expert Committee and national nomenclature groups to standardize drug ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Urea Cycle Disorder
Urea, also known as carbamide, is an organic compound with chemical formula . This amide has two amino groups (–) joined by a carbonyl functional group (–C(=O)–). It is thus the simplest amide of carbamic acid. Urea serves an important role in the metabolism of nitrogen-containing compounds by animals and is the main nitrogen-containing substance in the urine of mammals. It is a colorless, odorless solid, highly soluble in water, and practically non-toxic ( is 15 g/kg for rats). Dissolved in water, it is neither acidic nor alkaline. The body uses it in many processes, most notably nitrogen excretion. The liver forms it by combining two ammonia molecules () with a carbon dioxide () molecule in the urea cycle. Urea is widely used in fertilizers as a source of nitrogen (N) and is an important raw material for the chemical industry. In 1828 Friedrich Wöhler discovered that urea can be produced from inorganic starting materials, which was an important conceptual milestone i ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Ammonia
Ammonia is an inorganic compound of nitrogen and hydrogen with the formula . A stable binary hydride, and the simplest pnictogen hydride, ammonia is a colourless gas with a distinct pungent smell. Biologically, it is a common nitrogenous waste, particularly among aquatic organisms, and it contributes significantly to the nutritional needs of terrestrial organisms by serving as a precursor to 45% of the world's food and fertilizers. Around 70% of ammonia is used to make fertilisers in various forms and composition, such as urea and Diammonium phosphate. Ammonia in pure form is also applied directly into the soil. Ammonia, either directly or indirectly, is also a building block for the synthesis of many pharmaceutical products and is used in many commercial cleaning products. It is mainly collected by downward displacement of both air and water. Although common in nature—both terrestrially and in the outer planets of the Solar System—and in wide use, ammonia is both caust ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Hyperion Therapeutics
Hyperion may refer to: Greek mythology * Hyperion (Titan), one of the twelve Titans * ''Hyperion'', a byname of the Sun, Helios * Hyperion of Troy or Yperion, son of King Priam Science * Hyperion (moon), a moon of the planet Saturn * ''Hyperion'' (beetle), a genus of beetles in the family Carabidae * Hyperion (tree), a coast redwood in Northern California and the world's tallest known living tree * Hyperion proto-supercluster, a supercluster of galaxy groups discovered in 2018 * Project Hyperion (interstellar), preliminary study of a crewed interstellar starship or generation ship Literature * ''Hyperion'' (Hölderlin novel), a 1799 book by Friedrich Hölderlin * ''Hyperion'' (poem), a 1819 poem by John Keats * ''Hyperion'' (Longfellow novel), an 1839 book by Henry Wadsworth Longfellow * ''Hyperion'' (Simmons novel), a 1989 novel by Dan Simmons ** ''Hyperion Cantos'', the series of novels that started with ''Hyperion'' * ''Hyperion'' (magazine), a 1908–1910 Ge ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Buphenyl
Sodium phenylbutyrate, sold under the brand name Buphenyl among others, is a salt of an aromatic fatty acid, 4-phenylbutyrate (4-PBA) or 4-phenylbutyric acid. The compound is used to treat urea cycle disorders, because its metabolites offer an alternative pathway to the urea cycle to allow excretion of excess nitrogen. Sodium phenylbutyrate is also a histone deacetylase inhibitor and chemical chaperone, leading respectively to research into its use as an anti-cancer agent and in protein misfolding diseases such as cystic fibrosis. Structure and properties Sodium phenylbutyrate is a sodium salt of an aromatic fatty acid, made up of an aromatic ring and butyric acid. The chemical name for sodium phenylbutyrate is 4-phenylbutyric acid, sodium salt. It forms water-soluble off-white crystals. Uses Medical uses Sodium phenylbutyrate is taken orally or by nasogastric intubation as a tablet or powder, and tastes very salty and bitter. It treats urea cycle disorders, genetic diseas ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Horizon Pharma
Horizon Therapeutics Public Ltd Co is a biopharmaceutical company focused on researching, developing, and commercializing medicines that address critical needs for people impacted by rare and rheumatic diseases. Horizon primarily markets products in the United States, which represented 97% of Horizon's 2019 worldwide sales. In 2014 Horizon executed a tax inversion to move its legal headquarters to Ireland to avail of Ireland's low tax rates and beneficial corporate tax system. On May 2, 2019, shareholders of the company approved the change of the company's name to Horizon Therapeutics plc. Horizon's drug portfolio is focused on treatments for thyroid eye disease (TED), gout, rare diseases, and inflammatory diseases. Horizon's portfolio includes Tepezza (teprotumumab), Krystexxa (pegloticase), Actimmune (interferon gamma), Duexis (ibuprofen + famotidine), Pennsaid (diclofenac, 2% topical), Rayos/Lodotra (prednisone), Vimovo (naproxen/ esomeprazole magnesium), Buphenyl (sodium p ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
CYP2D6 Inhibitors
Cytochrome P450 2D6 (CYP2D6) is an enzyme that in humans is encoded by the ''CYP2D6'' gene. ''CYP2D6'' is primarily expressed in the liver. It is also highly expressed in areas of the central nervous system, including the substantia nigra. CYP2D6, a member of the cytochrome P450 mixed-function oxidase system, is one of the most important enzymes involved in the metabolism of xenobiotics in the body. In particular, CYP2D6 is responsible for the metabolism and clearance (medicine), elimination of approximately 25% of clinically used drugs, via the addition or removal of certain functional groups – specifically, hydroxylation, demethylation, and dealkylation. CYP2D6 also activates some prodrugs. This enzyme also metabolizes several endogenous substances, such as serotonin, hydroxytryptamines, neurosteroids, and both m-tyramine, ''m''-tyramine and tyramine, ''p''-tyramine which CYP2D6 metabolizes into dopamine in the brain and liver. Considerable variation exists in the effic ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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