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Fedratinib
Fedratinib, sold under the brand name Inrebic, is an anti-cancer medication used to treat myeloproliferative diseases including myelofibrosis. It is used in the form of fedratinib hydrochloride capsules that are taken by mouth. It is a semi-selective inhibitor of Janus kinase 2 (JAK-2). It was approved by the FDA on 16 August 2019. Myelofibrosis is a myeloid cancer associated with anemia, splenomegaly, and constitutional symptoms. Patients with myelofibrosis frequently harbor mutations which activate the JAK-STAT signaling pathway and which are sensitive to fedratinib. Phase I trial results focused on safety and efficacy of fedratinib in patients with high- or intermediate-risk primary or post–polycythemia vera/essential thrombocythemia myelofibrosis have been published in 2011. Medical uses In the United States, fedratinib is indicated for the treatment of adults with intermediate-2 or high-risk primary or secondary (following polycythemia vera or essential thrombocythem ...
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Myelofibrosis
Primary myelofibrosis (PMF) is a rare bone marrow blood cancer. It is classified by the World Health Organization (WHO) as a type of myeloproliferative neoplasm, a group of cancers in which there is activation and growth of mutated cells in the bone marrow. This is most often associated with a somatic mutation in the '' JAK2'', '' CALR'', or '' MPL'' genes. In PMF, the bony aspects of bone marrow are remodeled in a process called osteosclerosis; in addition, fibroblasts secrete collagen and reticulin proteins that are collectively referred to as fibrosis. These two pathological processes compromise the normal function of bone marrow, resulting in decreased production of blood cells such as erythrocytes (red cells), granulocytes, and megakaryocytes. The latter are responsible for the production of platelets. Signs and symptoms include fever, night sweats, bone pain, fatigue, and abdominal pain. Increased infections, bleeding and an enlarged spleen ( splenomegaly) are ...
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Celgene
Celgene Corporation, headquartered in Summit, New Jersey, was a pharmaceutical company that produced cancer and immunology drugs. Its primary products were Revlimid (lenalidomide), which is used in the treatment of multiple myeloma (63% of 2018 revenues); Pomalyst and Imnovid (Pomalidomide), also used in the treatment of multiple myeloma (13% of 2018 revenues); and Otezla (Apremilast), used in the treatment of psoriasis (11% of 2018 revenues). In 2018, 66% of the company's revenues came from the United States. In 2019, the company was acquired by Bristol Myers Squibb (BMS); as part of the acquisition, Otezla was sold to Amgen. History Celgene was originally a unit of Celanese. In 1986, Celanese completed the corporate spin-off of Celgene following the merger of Celanese with Hoechst AG, American Hoechst. In August 2000, Celgene acquired Signal Pharmaceuticals, Inc., a privately held company that developed pharmaceuticals to regulate disease-related genes. Signal Pharmaceuticals w ...
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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 ...
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Sanofi-Aventis
Sanofi S.A. is a French multinational pharmaceutical and healthcare company headquartered in Paris, France. The corporation was established in 1973 and merged with Synthélabo in 1999 to form Sanofi-Synthélabo. In 2004, Sanofi-Synthélabo merged with Aventis and renamed to Sanofi-Aventis, which were each the product of several previous mergers. It changed its name back to Sanofi in May 2011. The company trades as "SAN" on Euronext Paris and "SNY" on Nasdaq in the United States, and is a component of the Euro Stoxx 50 stock market index. In 2023, the company’s seat in Forbes Global 2000 was 89. Sanofi engages in the research and development, manufacturing, and marketing of pharmacological products, principally in the prescription market, but the firm also develops over-the-counter medications. The corporation covers seven major therapeutic areas: cardiovascular, central nervous system, diabetes, internal medicine, oncology, thrombosis, and vaccines. It is the world's ...
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Pyrimidines
Pyrimidine (; ) is an aromatic, heterocyclic, organic compound similar to pyridine (). One of the three diazines (six-membered heterocyclics with two nitrogen atoms in the ring), it has nitrogen atoms at positions 1 and 3 in the ring. The other diazines are pyrazine (nitrogen atoms at the 1 and 4 positions) and pyridazine (nitrogen atoms at the 1 and 2 positions). In nucleic acids, three types of nucleobases are pyrimidine derivatives: cytosine (C), thymine (T), and uracil (U). Occurrence and history The pyrimidine ring system has wide occurrence in nature as substituted and ring fused compounds and derivatives, including the nucleotides cytosine, thymine and uracil, thiamine (vitamin B1) and alloxan. It is also found in many synthetic compounds such as barbiturates and the HIV drug zidovudine. Although pyrimidine derivatives such as alloxan were known in the early 19th century, a laboratory synthesis of a pyrimidine was not carried out until 1879, when Grimaux reported the ...
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Tert-butyl Compounds
In organic chemistry, butyl is a four-carbon alkyl radical or substituent group with general chemical formula , derived from either of the two isomers (''n''-butane and isobutane) of butane. The isomer ''n''-butane can connect in two ways, giving rise to two "-butyl" groups: * If it connects at one of the two terminal carbon atoms, it is normal butyl or ''n''-butyl: (preferred IUPAC name: butyl) * If it connects at one of the non-terminal (internal) carbon atoms, it is secondary butyl or ''sec''-butyl: (preferred IUPAC name: butan-2-yl) The second isomer of butane, isobutane, can also connect in two ways, giving rise to two additional groups: * If it connects at one of the three terminal carbons, it is isobutyl: (preferred IUPAC name: 2-methylpropyl) * If it connects at the central carbon, it is tertiary butyl, ''tert''-butyl or ''t''-butyl: (preferred IUPAC name: ''tert''-butyl) Nomenclature According to IUPAC nomenclature, "isobutyl", "''sec''-butyl", and "''tert''- ...
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Sulfonamides
In organic chemistry, the sulfonamide functional group (also spelled sulphonamide) is an organosulfur group with the Chemical structure, structure . It consists of a sulfonyl group () connected to an amine group (). Relatively speaking this group is unreactive. Because of the rigidity of the functional group, sulfonamides are typically crystalline; for this reason, the formation of a sulfonamide is a classic method to convert an amine into a crystalline derivative which can be identified by its melting point. Many important drugs contain the sulfonamide group. A sulfonamide (compound) is a chemical compound that contains this group. The general formula is or , where each R is some organic group; for example, "methanesulfonamide" (where R = methane, R' = R" = hydrogen) is . Any sulfonamide can be considered as derived from a sulfonic acid by replacing a hydroxyl group () with an amine group. In medicine, the term "sulfonamide" is sometimes used as a synonym for Sulfonamide (m ...
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Protein Kinase Inhibitors
A protein kinase inhibitor (PKI) is a type of enzyme inhibitor that blocks the action of one or more protein kinases. Protein kinases are enzymes that phosphorylate (add a phosphate, or PO4, group) to a protein and can modulate its function. The phosphate groups are usually added to serine, threonine, or tyrosine amino acids on the protein. Most kinases act on both serine and threonine, the tyrosine kinases act on tyrosine, and a number ( dual-specificity kinases) act on all three. There are also protein kinases that phosphorylate other amino acids, including histidine kinases that phosphorylate histidine residues. Phosphorylation regulates many biological processes, and protein kinase inhibitors can be used to treat diseases due to hyperactive protein kinases (including mutant or overexpressed kinases in cancer) or to modulate cell functions to overcome other disease drivers. Clinical use Kinase inhibitors such as dasatinib are often used in the treatment of cancer and inflamm ...
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Orphan Drugs
An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation (if there is any) of the country. Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development. Examples of this can be that in the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs ...
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Cancer Treatments
Cancer treatments are a wide range of treatments available for the many different types of cancer, with each cancer type needing its own specific treatment. Treatments can include surgery, chemotherapy, radiation therapy, hormonal therapy, targeted therapy including small-molecule drugs or monoclonal antibodies, and PARP inhibitors such as olaparib. Other therapies include hyperthermia, immunotherapy, photodynamic therapy, and stem-cell therapy. Most commonly cancer treatment involves a series of separate therapies such as chemotherapy before surgery. Angiogenesis inhibitors are sometimes used to enhance the effects of immunotherapies. The choice of therapy depends upon the location and grade of the tumor and the stage of the disease, as well as the general state of the patient. Biomarker testing can help to determine the type of cancer, and indicate the best therapy. A number of experimental cancer treatments are continuously under development. In 2023 it was estimated ...
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Drugs Developed By Bristol Myers Squibb
A drug is any chemical substance other than a nutrient or an essential dietary ingredient, which, when administered to a living organism, produces a biological effect. Consumption of drugs can be via insufflation (medicine), inhalation, drug injection, injection, smoking, ingestion, absorption (skin), absorption via a dermal patch, patch on the skin, suppository, or sublingual administration, dissolution under the tongue. In pharmacology, a drug is a chemical substance, typically of known structure, which, when administered to a living organism, produces a biological effect. A pharmaceutical drug, also called a medication or medicine, is a chemical substance used to pharmacotherapy, treat, cure, preventive healthcare, prevent, or medical diagnosis, diagnose a disease or to promote well-being. Traditionally drugs were obtained through extraction from medicinal plants, but more recently also by organic synthesis. Pharmaceutical drugs may be used for a limited duration, or on a re ...
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