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Glycerol Phenylbutyrate
Glycerol phenylbutyrate, sold under the brand name Ravicti, is a nitrogen-binding agent 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 was developed by Hyperion Therapeutics based on the existing medication sodium phenylbutyrate, and received approval in February 2013. Medical uses Glycerol phenylbutyrate is indicated In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis ... for chronic management of people with urea cycle disorders who cannot be managed by dietary protein restriction and/or amino acid supplementation alone. Society and culture Economics Hyperion has been criticized for setting a high price for the drug. ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Oral Administration
Oral administration is a route of administration whereby a substance is taken through the Human mouth, mouth, swallowed, and then processed via the digestive system. This is a common route of administration for many medications. Oral administration can be easier and less painful than other routes of administration, such as Injection (medicine), 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 able to swallow. Terminology ''Per os'' (; ''P.O.'') is an adverbial phrase meaning literally from Latin "through the mouth" or "by mouth". The expression is used in medicine to describe a treatment that is taken orally (but not ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Health Canada
Health Canada (HC; )Health Canada is the applied title under the Federal Identity Program; the legal title is Department of Health (). is the Structure of the Canadian federal government#Departments, with subsidiary units, department of the Government of Canada responsible for national health policy. The department itself is also responsible for numerous federal health-related agencies, including the Canadian Food Inspection Agency (CFIA) and the Public Health Agency of Canada (PHAC), among others. These organizations help to ensure compliance with federal law in a variety of Healthcare in Canada, healthcare, Agriculture in Canada, agricultural, and Pharmaceutics, pharmaceutical activities. This responsibility also involves extensive collaboration with various other federal- and provincial-level organizations in order to ensure the safety of food, health, and Medication, pharmaceutical products—including the regulation of health research and pharmaceutical manufacturing/Clinical ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Medication
Medication (also called medicament, medicine, pharmaceutical drug, medicinal product, medicinal drug or simply drug) is a drug used to medical diagnosis, diagnose, cure, treat, or preventive medicine, prevent disease. Drug therapy (pharmacotherapy) is an important part of the medicine, medical field and relies on the science of pharmacology for continual advancement and on pharmacy for appropriate management. Drugs are Drug class, classified in many ways. One of the key divisions is by level of controlled substance, control, which distinguishes prescription drugs (those that a pharmacist dispenses only on the medical prescription) from over-the-counter drugs (those that consumers can order for themselves). Medicines may be classified by mode of action, route of administration, biological system affected, or therapeutic effects. The World Health Organization keeps a list of essential medicines. Drug discovery and drug development are complex and expensive endeavors undertake ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Urea Cycle Disorder
The urea cycle (also known as the ornithine cycle) is a cycle of biochemical reactions that produces urea (NH2)2CO from ammonia (NH3). Animals that use this cycle, mainly amphibians and mammals, are called ureotelic. The urea cycle converts highly toxic ammonia to urea for excretion. This cycle was the first metabolic cycle to be discovered by Hans Krebs and Kurt Henseleit in 1932, five years before the discovery of the TCA cycle. The urea cycle was described in more detail later on by Ratner and Cohen. The urea cycle takes place primarily in the liver and, to a lesser extent, in the kidneys. Function Amino acid catabolism results in waste ammonia. All animals need a way to excrete this product. Most aquatic organisms, or ammonotelic organisms, excrete ammonia without converting it. Organisms that cannot easily and safely remove nitrogen as ammonia convert it to a less toxic substance, such as urea, via the urea cycle, which occurs mainly in the liver. Urea produced by the liv ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Ammonia
Ammonia is an inorganic chemical compound of nitrogen and hydrogen with the chemical formula, formula . A Binary compounds of hydrogen, stable binary hydride and the simplest pnictogen hydride, ammonia is a colourless gas with a distinctive pungent smell. It is widely used in fertilizers, refrigerants, explosives, cleaning agents, and is a precursor for numeous chemicals. Biologically, it is a common nitrogenous waste, and it contributes significantly to the nutritional needs of terrestrial organisms by serving as a precursor to fertilisers. Around 70% of ammonia produced industrially 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 chemicals. In many countries, it is classified as an List of extremely hazardous substances, extremely hazardous substance. Ammonia is toxic, cau ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Food And Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, Prescription drug, prescription and Over-the-counter drug, over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, Animal feed, animal foods & feed and Veterinary medicine, veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C). However, the agency also enforces other laws, notably Section 361 of the Public Health Service Act as well as associated regulations. Much of this regulatory-enforcement work is ... [...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 ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Sodium Phenylbutyrate
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 diseases ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Indicated
In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis is the assessment that a particular medical condition is present while an indication is a reason for use. The opposite of an indication is a contraindication, a reason to withhold a certain medical treatment because the risks of treatment clearly outweigh the benefits. In the United States, indications for prescription drugs are approved by the FDA. Indications are included in the Indications and Usage section of the Prescribing Information. The primary role of this section of labeling is to enable health care practitioners to readily identify appropriate therapies for patients by clearly communicating the drug's approved indication(s). The Indications and Usage section states the disease or condition, or manifestation or symptoms thereof ... [...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 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 N,N-Dimethyltryptamine, hydroxytryptamines, neurosteroids, and both ''m''-tyramine and ''p''-tyramine which CYP2D6 metabolizes into dopamine in the brain and liver. Considerable variation exists in the efficiency and amount of C ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
