Sonidegib
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Sonidegib
Sonidegib (INN), sold under the brand name Odomzo, is a medication used to treat cancer. Sonidegib is Hedgehog signaling pathway inhibitor (via smoothened antagonism). Approvals and indications It was approved for medical use in the United States and in the European Union in 2015 It is indicated for the treatment of adults with locally advanced basal-cell carcinoma that has recurred following surgery or radiation therapy, or those who are not candidates for surgery or radiation therapy. Pharmacology Sonidegib is administered by mouth. Common side effects include muscle spasms, hair loss, fatigue, abdominal pain, nausea, headache, and weight loss. Sonidegib binds to and inhibits smoothened to inhibit activation of the Hedgehog pathway. Sonidegib is primarily metabolized by CYP3A and is eliminated hepatically. Development It has been investigated as a potential treatment for: * Pancreatic cancer * Breast cancer * Basal cell carcinoma of the skin * Small cell lung cancer * Med ...
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Basal Cell Carcinoma
Basal-cell carcinoma (BCC), also known as basal-cell cancer, is the most common type of skin cancer. It often appears as a painless raised area of skin, which may be shiny with small blood vessels running over it. It may also present as a raised area with ulceration. Basal-cell cancer grows slowly and can damage the tissue around it, but it is unlikely to spread to distant areas or result in death. Risk factors include exposure to ultraviolet light, having lighter skin, radiation therapy, long-term exposure to arsenic and poor immune-system function. Exposure to UV light during childhood is particularly harmful. Tanning beds have become another common source of ultraviolet radiation. Diagnosis often depends on skin examination, confirmed by tissue biopsy. It remains unclear whether sunscreen affects the risk of basal-cell cancer. Treatment is typically by surgical removal. This can be by simple excision if the cancer is small; otherwise, Mohs surgery is generally recomme ...
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Hedgehog Signaling Pathway
The Hedgehog signaling pathway is a signaling pathway that transmits information to embryonic cells required for proper cell differentiation. Different parts of the embryo have different concentrations of hedgehog signaling proteins. The pathway also has roles in the adult. Diseases associated with the malfunction of this pathway include cancer. The Hedgehog signaling pathway is one of the key regulators of animal development and is present in all bilaterians. The pathway takes its name from its polypeptide ligand, an intracellular signaling molecule called Hedgehog (''Hh'') found in fruit flies of the genus ''Drosophila''; fruit fly larva lacking the ''Hh'' gene are said to resemble hedgehogs. ''Hh'' is one of Drosophila's segment polarity gene products, involved in establishing the basis of the fly body plan. Larvae without ''Hh'' are short and spiny, resembling the hedgehog animal. The molecule remains important during later stages of embryogenesis and metamorphosis. Mamm ...
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Novartis Brands
Novartis AG is a Swiss-American multinational corporation, multinational pharmaceutical company, pharmaceutical corporation based in Basel, Switzerland and Cambridge, Massachusetts, United States (global research).name="novartis.com">https://www.novartis.com/research-development/research-locations It is one of the largest pharmaceutical companies in the world. Novartis manufactures the drugs clozapine (Clozaril), diclofenac (Voltaren; sold to GlaxoSmithKline in 2015 deal), carbamazepine (Tegretol), valsartan (Diovan), imatinib mesylate (Gleevec/Glivec), cyclosporine (Neoral/Sandimmune), letrozole (Femara), methylphenidate (Ritalin; production ceased 2020), terbinafine (Lamisil), deferasirox (Exjade), and others. In March 1996, the companies Ciba-Geigy and Sandoz merged to form Novartis; the pharmaceutical and agrochemical divisions of both companies formed Novartis as an independent entity. Other Ciba-Geigy and Sandoz businesses were sold, or, like Ciba Specialty Chemicals, sp ...
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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 mo ...
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Glioblastoma Multiforme
Glioblastoma, previously known as glioblastoma multiforme (GBM), is one of the most aggressive types of cancer that begin within the brain. Initially, signs and symptoms of glioblastoma are nonspecific. They may include headaches, personality changes, nausea, and symptoms similar to those of a stroke. Symptoms often worsen rapidly and may progress to unconsciousness. The cause of most cases of glioblastoma is not known. Uncommon risk factors include genetic disorders, such as neurofibromatosis and Li–Fraumeni syndrome, and previous radiation therapy. Glioblastomas represent 15% of all brain tumors. They can either start from normal brain cells or develop from an existing low-grade astrocytoma. The diagnosis typically is made by a combination of a CT scan, MRI scan, and tissue biopsy. There is no known method of preventing the cancer. Treatment usually involves surgery, after which chemotherapy and radiation therapy are used. The medication temozolomide is frequently used as ...
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Trifluoromethyl Ethers
The trifluoromethyl group is a functional group that has the formula -CF3. The naming of is group is derived from the methyl group (which has the formula -CH3), by replacing each hydrogen atom by a fluorine atom. Some common examples are trifluoromethane H–, 1,1,1-trifluoroethane –, and hexafluoroacetone –CO–. Compounds with this group are a subclass of the organofluorines. Properties The trifluoromethyl group has a significant electronegativity that is often described as being intermediate between the electronegativities of fluorine and chlorine. For this reason, trifluoromethyl-substituted compounds are often strong acids, such as trifluoromethanesulfonic acid and trifluoroacetic acid. Conversely, the trifluoromethyl group lowers the basicity of compounds like trifluoroethanol. Uses The trifluoromethyl group occurs in certain pharmaceuticals, drugs, and abiotically synthesized natural fluorocarbon based compounds. The medicinal use of the trifloromethyl group dates f ...
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Aminopyridines
Aminopyridine may refer to any of several chemical compounds: * 2-Aminopyridine * 3-Aminopyridine 3-Aminopyridine is an aminopyridine. It is a colorless solid. Preparation 3-Aminopyridine is prepared by heating nicotinamide with sodium hypobromite which is in turn prepared ''in situ'' by the reaction of sodium hydroxide and bromine at 70&nb ... * 4-Aminopyridine (4-AP), also known as fampridine or dalfampridine {{Short pages monitor ...
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Morpholines
Morpholine is an organic chemical compound having the chemical formula O( C H2CH2)2 NH. This heterocycle features both amine and ether functional groups. Because of the amine, morpholine is a base; its conjugate acid is called morpholinium. For example, treating morpholine with hydrochloric acid makes the salt morpholinium chloride. It is a colorless liquid with a weak, ammonia- or fish-like odor. The naming of morpholine is attributed to Ludwig Knorr, who incorrectly believed it to be part of the structure of morphine. Production Morpholine is often produced industrially by the dehydration of diethanolamine with sulfuric acid: : Uses Industrial applications Morpholine is a common additive, in parts per million concentrations, for pH adjustment in both fossil fuel and nuclear power plant steam systems. Morpholine is used because its volatility is about the same as water, so once it is added to the water, its concentration becomes distributed rather evenly in both the water a ...
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Benzamides
Benzamide is a organic compound with the chemical formula of C6H5C(O)NH2. It is the simplest amide derivative of benzoic acid. In powdered form, it appears as a white solid, while in crystalline form, it appears as colourless crystals. It is slightly soluble in water, and soluble in many organic solvents. It is a natural alkaloid found in the herbs of Berberis pruinosa. Chemical derivatives A number of substituted A substitution reaction (also known as single displacement reaction or single substitution reaction) is a chemical reaction during which one functional group in a chemical compound is replaced by another functional group. Substitution reactions ar ... benzamides are commercial drugs, including: See also * References External links Physical characteristics {{Authority control Phenyl compounds ...
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Essential Thrombocythaemia
Essential thrombocythemia (ET) is a rare chronic blood cancer (myeloproliferative neoplasm) characterised by the overproduction of platelets (thrombocytes) by megakaryocytes in the bone marrow. It may, albeit rarely, develop into acute myeloid leukemia or myelofibrosis. It is a type of myeloproliferative neoplasm (blood cancers) wherein the body makes too many white or red blood cells, or platelets). Signs and symptoms Most people with essential thrombocythemia are without symptoms at the time of diagnosis, which is usually made after noting an elevated platelet level on a routine complete blood count (CBC). The most common symptoms are bleeding (due to dysfunctional platelets), blood clots (e.g., deep vein thrombosis or pulmonary embolism), fatigue, headache, nausea, vomiting, abdominal pain, visual disturbances, dizziness, fainting, and numbness in the extremities; the most common signs are increased white blood cell count, reduced red blood cell count, and an enlarged sple ...
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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 growth of abnormal cells in the bone marrow. This is most often associated with a somatic mutation in the JAK2, CALR, or MPL gene markers. In PMF, the healthy marrow is replaced by scar tissue (fibrosis), resulting in a lack of production of normal blood cells. Symptoms include anemia, increased infection and an enlarged spleen ( splenomegaly). In 2016, prefibrotic primary myelofibrosis was formally classified as a distinct condition that progresses to overt PMF in many patients, the primary diagnostic difference being the grade of fibrosis. Signs and symptoms The primary feature of primary myelofibrosis is bone marrow fibrosis, but it is often accompanied by: * Abdominal fullness related to an enlarged spleen (splenomegaly). * Bone pain * Bruising and easy bleeding due to inad ...
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Chronic Myeloid Leukemia
Chronic myelogenous leukemia (CML), also known as chronic myeloid leukemia, is a cancer of the white blood cells. It is a form of leukemia characterized by the increased and unregulated growth of myeloid cells in the bone marrow and the accumulation of these cells in the blood. CML is a clonal bone marrow stem cell disorder in which a proliferation of mature granulocytes (neutrophils, eosinophils and basophils) and their precursors is found. It is a type of myeloproliferative neoplasm associated with a characteristic chromosomal translocation called the Philadelphia chromosome. CML is largely treated with targeted drugs called tyrosine-kinase inhibitors (TKIs) which have led to dramatically improved long-term survival rates since 2001. These drugs have revolutionized treatment of this disease and allow most patients to have a good quality of life when compared to the former chemotherapy drugs. In Western countries, CML accounts for 15–25% of all adult leukemias and 14% of leuk ...
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