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Enzomenib
Enzomenib is an investigational new drug that is being evaluated for the treatment of acute leukemia. It is a small molecule inhibitor that targets the interaction between menin and mixed-lineage leukemia (MLL) proteins. Enzomenib particularly in patients with KMT2A (MLL) rearrangements or NPM1 mutations. The U.S. Food and Drug Administration (FDA) has granted both Fast Track and Orphan Drug An orphan drug is a medication, 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 th ... designations to Enzomenib. References {{antineoplastic-drug-stub Alkenes Antineoplastic drugs Azetidines Benzamides Carboxamides Ethers Fluorobenzenes Isopropylamino compounds Piperidines Pyrimidines Spiro compounds ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Investigational New Drug
The United States Food and Drug Administration's Investigational New Drug (IND) program is the means by which a pharmaceutical industry, pharmaceutical company obtains permission to start human clinical trials and to ship an experimental drug interstate commerce, across state lines (usually to clinical investigators) before a marketing application for the drug has been approved. Regulations are primarily at . Similar procedures are followed in the European Union, Japan, and Canada due to regulatory harmonization efforts by the International Council for Harmonisation of Technical Requirements for Pharmaceuticals for Human Use, International Council for Harmonisation. Types * Research or investigator INDs are non-commercial INDs filed by researchers to study an unapproved drug or to study an approved drug for a new indication or in a new patient population. * Expanded access, Emergency Use INDs, also called compassionate use or single-patient INDs, are filed for emergency use of a ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
MEN1
Menin is a protein that in humans is encoded by the ''MEN1'' gene. Menin is a putative tumor suppressor associated with multiple endocrine neoplasia type 1 (MEN-1 syndrome) and has autosomal dominant inheritance. Variations in the MEN1 gene can cause pituitary adenomas, hyperparathyroidism, pancreatic neuroendocrine tumors, gastrinoma, and adrenocortical cancers. ''In vitro'' studies have shown that menin is localized to the nucleus, possesses two functional nuclear localization signals, and inhibits transcriptional activation by JunD. However, the function of this protein is not known. Two messages have been detected on northern blots but the larger message has not been characterized. Two variants of the shorter transcript have been identified where alternative splicing affects the coding sequence. Five variants where alternative splicing takes place in the 5' UTR have also been identified. History In 1988, researchers at Uppsala University Hospital and the Karolinska I ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
KMT2A
Histone-lysine ''N''-methyltransferase 2A, also known as acute lymphoblastic leukemia 1 (ALL-1), myeloid/lymphoid or mixed-lineage leukemia 1 (MLL1), or zinc finger protein HRX (HRX), is an enzyme that in humans is encoded by the ''KMT2A'' gene. MLL1 is a histone methyltransferase deemed a positive global regulator of gene transcription. This protein belongs to the group of histone-modifying enzymes comprising transactivation domain 9aaTAD; ; and is involved in the epigenetic maintenance of transcriptional memory. Its role as an epigenetic regulator of neuronal function is an ongoing area of research. Function Transcriptional regulation KMT2A gene encodes a transcriptional coactivator that plays an essential role in regulating gene expression during early development and hematopoiesis. The encoded protein contains multiple conserved functional domains. One of these domains, the SET domain, is responsible for its histone H3 lysine 4 (H3K4) methyltransferase activity whi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Fast Track (FDA)
Fast track is a designation by the United States Food and Drug Administration (FDA) of an investigational drug for expedited review to facilitate development of drugs that treat a serious or life-threatening condition and fill an unmet medical need. Fast track designation must be requested by the drug company. The request can be initiated at any time during the drug development process. FDA will review the request and attempt to make a decision within sixty days. Purpose Fast track is one of five FDA approaches to make new drugs available as rapidly as possible: the others are priority review, breakthrough therapy, accelerated approval and regenerative medicine advanced therapy. Fast track was introduced by the FDA Modernization Act of 1997. Requirements Fast track designation is designed to aid in the development and expedite the review of drugs which show promise in treating a serious or life-threatening disease and address an unmet medical need. Serious condition: det ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drug Act Of 1983
The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's disease, myoclonus, ALS, Tourette syndrome or muscular dystrophy which affect small numbers of individuals residing in the United States. Orphan drug designation does not indicate that the therapeutic is either safe and effective or legal to manufacture and market in the United States. That process is handled through other offices in the US Food and Drug Administration. Instead, the designation means only that the sponsor qualifies for certain benefits from the federal government, such as market exclusivity and reduced taxes. In 1982 an informal coalition of supporters and families of patients with rare diseases who formed National Organization for Rare Disorders (NORD) and others, called for change to legislation to support development of orphan drugs, or drugs for treating rare diseases. They succeeded in getting the United S ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Alkenes
In organic chemistry, an alkene, or olefin, is a hydrocarbon containing a carbon–carbon double bond. The double bond may be internal or at the terminal position. Terminal alkenes are also known as Alpha-olefin, α-olefins. The International Union of Pure and Applied Chemistry (IUPAC) Preferred IUPAC name, recommends using the name "alkene" only for Open-chain compound, acyclic hydrocarbons with just one double bond; alkadiene, alkatriene, etc., or polyene for acyclic hydrocarbons with two or more double bonds; cycloalkene, cycloalkadiene, etc. for Cyclic compound, cyclic ones; and "olefin" for the general class – cyclic or acyclic, with one or more double bonds. Acyclic alkenes, with only one double bond and no other functional groups (also known as mono-enes) form a homologous series of hydrocarbons with the general formula with ''n'' being a >1 natural number (which is two hydrogens less than the corresponding alkane). When ''n'' is four or more, isomers are possible, ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Antineoplastic Drugs
Chemotherapy (often abbreviated chemo, sometimes CTX and CTx) is the type of cancer treatment that uses one or more anti-cancer drugs ( chemotherapeutic agents or alkylating agents) in a standard regimen A regimen is a plan, or course of action such as a Diet (nutrition), diet, exercise or medical treatment. A salt#Health effects, low-salt diet is a regimen. A course of penicillin is a regimen, and there are many chemotherapy regimens in the trea .... Chemotherapy may be given with a curative intent (which almost always involves combinations of drugs), or it may aim only to prolong life or to Palliative care, reduce symptoms (Palliative care, palliative chemotherapy). Chemotherapy is one of the major categories of the medical discipline specifically devoted to pharmacotherapy for cancer, which is called ''oncology#Specialties, medical oncology''. The term ''chemotherapy'' now means the non-specific use of intracellular poisons to inhibit mitosis (cell division) or to indu ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Azetidines
Azetidine is a Saturated and unsaturated compounds, saturated heterocyclic organic compound containing three carbon atoms and one nitrogen atom. It is a liquid at room temperature with a strong odor of ammonia and is strongly Base (chemistry)#Strong bases, basic compared to most secondary amines. Synthesis and occurrence Azetidines can be prepared by reduction of azetidinones (β-lactams) with lithium aluminium hydride. Even more effective is a mixture of lithium aluminium hydride and aluminium trichloride, a source of "AlClH2" and "AlCl2H". Azetidine can also be produced by a multistep route from 3-Amino-1-propanol, 3-amino-1-propanol. Regio- and diastereoselective synthesis of 2-arylazetidines could be performed from appropriately substituted oxiranes via ring transformation. It is controlled by Baldwin's Rules with remarkable functional group tolerance. The Paternò−Büchi reaction has Aza Paternò−Büchi reaction, an ''aza'' analogue. Azetidine and its derivatives ar ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Benzamides
Benzamide is an organic compound with the chemical formula of C7H7NO. 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 Alkaloids are a broad class of natural product, naturally occurring organic compounds that contain at least one nitrogen atom. Some synthetic compounds of similar structure may also be termed alkaloids. Alkaloids are produced by a large varie ... found in the herbs of '' Berberis pruinosa''. Chemical derivatives A number of substituted benzamides are commercial drugs, including: See also * References External links Physical characteristics {{Authority control Phenyl compounds ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Ethers
In organic chemistry, ethers are a class of compounds that contain an ether group, a single oxygen atom bonded to two separate carbon atoms, each part of an organyl group (e.g., alkyl or aryl). They have the general formula , where R and R′ represent the organyl groups. Ethers can again be classified into two varieties: if the organyl groups are the same on both sides of the oxygen atom, then it is a simple or symmetrical ether, whereas if they are different, the ethers are called mixed or unsymmetrical ethers. A typical example of the first group is the solvent and anaesthetic diethyl ether, commonly referred to simply as "ether" (). Ethers are common in organic chemistry and even more prevalent in biochemistry, as they are common linkages in carbohydrates and lignin. Structure and bonding Ethers feature bent linkages. In dimethyl ether, the bond angle is 111° and C–O distances are 141 pm. The barrier to rotation about the C–O bonds is low. The bonding of oxygen ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Fluorobenzenes
Fluorobenzenes are a group of aryl fluorides/halobenzenes consisting of one or more fluorine atoms as substituents on a benzene core. They have the formula C6H6–''n''F''n'', where ''n'' = 1–6 is the number of fluorine atoms. Depending on the number of fluorine substituents, there may be several constitutional isomers possible. * Monofluorobenzene * Difluorobenzene ** 1,2-Difluorobenzene ** 1,3-Difluorobenzene ** 1,4-Difluorobenzene * Trifluorobenzene ** 1,2,3-Trifluorobenzene ** 1,2,4-Trifluorobenzene ** 1,3,5-Trifluorobenzene * Tetrafluorobenzene ** 1,2,3,4-Tetrafluorobenzene ** 1,2,3,5-Tetrafluorobenzene ** 1,2,4,5-Tetrafluorobenzene * Pentafluorobenzene * Hexafluorobenzene See also *Chlorobenzenes *Bromobenzenes *Iodobenzenes Iodobenzenes are a group of aryl iodides/halobenzenes consisting of one or more iodine atoms as substituents on a benzene core. They have the formula C6H6–''n''I''n'', where ''n'' = 1–6 is the number of iodine atoms. Depending on the nu ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
