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Luspatercept-aamt
Luspatercept, sold under the brand name Reblozyl, is a medication used for the treatment of anemia in beta thalassemia and myelodysplastic syndromes. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses Luspatercept is indicated for the treatment of adults with transfusion-dependent anemia due to very low, low and intermediate-risk myelodysplastic syndromes (MDS) with ring sideroblasts, who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy. Luspatercept is indicated for the treatment of adults with transfusion-dependent anaemia associated with beta thalassaemia. Side effects Possible adverse effects include temporary bone pain, joint pains (arthralgias), dizziness, elevated blood pressure (hypertension) and elevated uric acid levels (hyperuricemia). There was also an increased risk of thrombosis (blood clots) in patients who have risk factors for thrombosis who are taking luspatercept. S ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Acceleron Pharma
Acceleron Pharma, Inc. is an American clinical stage biopharmaceutical company based in Cambridge, Massachusetts with a broad focus on developing medicines that regulate the transforming growth factor beta (TGF-β) superfamily of proteins, which play fundamental roles in the growth and repair of cells and tissues such as red blood cells, muscle, bone, and blood vessels. Pipeline Acceleron has four drugs in clinical trials, and one in preclinical development. * Luspatercept (ACE-536) for anemia * Sotatercept (ACE-011) for kidney disease * Dalantercept (ACE-041) for kidney cancer * ACE-083 for muscular disorders History The company was formed in June 2003 in Cambridge, Massachusetts as a Delaware corporation; the original name was Phoenix Pharma. The founders were scientists Jasbir Seehra, Tom Maniatis, Mark Ptashne, Wylie Vale, and scientific advisor Joan Massague, and business people and investors John Knopf and Christoph Westphal of Polaris Venture Partners, who served as f ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Subcutaneous Injection
Subcutaneous administration is the insertion of medications beneath the skin either by injection or infusion. A subcutaneous injection is administered as a bolus into the subcutis, the layer of skin directly below the dermis and epidermis, collectively referred to as the cutis. The instruments are usually a hypodermic needle and a syringe. Subcutaneous injections are highly effective in administering medications such as insulin, morphine, diacetylmorphine and goserelin. Subcutaneous administration may be abbreviated as SC, SQ, subcu, sub-Q, SubQ, or subcut. Subcut is the preferred abbreviation to reduce the risk of misunderstanding and potential errors. Subcutaneous tissue has few blood vessels and so drugs injected here are for slow, sustained rates of absorption, often with some amount of depot effect. Compared with other routes of administration, it is slower than intramuscular injections but still faster than intradermal injections. Subcutaneous infusion (as opposed ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Transforming Growth Factor Beta
Transforming growth factor beta (TGF-β) is a multifunctional cytokine belonging to the transforming growth factor superfamily that includes three different mammalian isoforms (TGF-β 1 to 3, HGNC symbols TGFB1, TGFB2, TGFB3) and many other signaling proteins. TGFB proteins are produced by all white blood cell lineages. Activated TGF-β complexes with other factors to form a serine/threonine kinase complex that binds to TGF-β receptors. TGF-β receptors are composed of both type 1 and type 2 receptor subunits. After the binding of TGF-β, the type 2 receptor kinase phosphorylates and activates the type 1 receptor kinase that activates a signaling cascade. This leads to the activation of different downstream substrates and regulatory proteins, inducing transcription of different target genes that function in differentiation, chemotaxis, proliferation, and activation of many immune cells. TGF-β is secreted by many cell types, including macrophages, in a latent form in which it ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Drugs Developed By Bristol Myers Squibb
A drug is any chemical substance that causes a change in an organism's physiology or psychology when consumed. Drugs are typically distinguished from food and substances that provide nutritional support. Consumption of drugs can be via inhalation, injection, smoking, ingestion, absorption via a patch on the skin, suppository, or 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 treat, cure, prevent, or 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 regular basis for chronic disorders. Pharmaceutical drugs are often classified into drug classes—groups of related drugs ... [...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 Food and Drug Administration (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 ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drug
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions 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 in many countries and has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development. 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 which would otherwise lack sufficient profit motive to attract corporate research budgets and personnel. Definition According to the US Food and Drug Administration (FDA), an orphan drug is defined as one "intended for ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
European Medicines Agency
The European Medicines Agency (EMA) is an agency of the European Union (EU) in charge of the evaluation and supervision of medicinal products. Prior to 2004, it was known as the European Agency for the Evaluation of Medicinal Products or European Medicines Evaluation Agency (EMEA).Set up by EC Regulation No. 2309/93 as the European Agency for the Evaluation of Medicinal Products, and renamed by EC Regulation No. 726/2004 to the European Medicines Agency, it had the acronym EMEA until December 2009. The European Medicines Agency does not call itself EMA either – it has no official acronym but may reconsider if EMA becomes commonly accepted (secommunication on new visual identity an). The EMA was set up in 1995, with funding from the European Union and the pharmaceutical industry, as well as indirect subsidy from member states, its stated intention to harmonise (but not replace) the work of existing national medicine regulatory bodies. The hope was that this plan would not onl ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Celgene
Celgene Corporation is a pharmaceutical company that makes cancer and immunology drugs. Its major product is Revlimid ( lenalidomide), which is used in the treatment of multiple myeloma, and also in certain anemias. The company is incorporated in Delaware, headquartered in Summit, New Jersey, and a subsidiary of Bristol Myers Squibb (BMS).Bristol-Myers Squibb Completes Acquisition of Celgene, Creating a Leading Biopharma Company PM BMS, November 20, 2019; retrieved May 20, 2020 History Celgene was originally a unit of . In 1986, Celanese co ...[...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Drug Development
Drug development is the process of bringing a new pharmaceutical drug to the market once a lead compound has been identified through the process of drug discovery. It includes preclinical research on microorganisms and animals, filing for regulatory status, such as via the United States Food and Drug Administration for an investigational new drug to initiate clinical trials on humans, and may include the step of obtaining regulatory approval with a new drug application to market the drug. The entire process – from concept through preclinical testing in the laboratory to clinical trial development, including Phase I–III trials – to approved vaccine or drug typically takes more than a decade. New chemical entity development Broadly, the process of drug development can be divided into preclinical and clinical work. Pre-clinical New chemical entities (NCEs, also known as new molecular entities or NMEs) are compounds that emerge from the process of drug discovery. Th ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Erythroid
Red blood cells (RBCs), also referred to as red cells, red blood corpuscles (in humans or other animals not having nucleus in red blood cells), haematids, erythroid cells or erythrocytes (from Greek ''erythros'' for "red" and ''kytos'' for "hollow vessel", with ''-cyte'' translated as "cell" in modern usage), are the most common type of blood cell and the vertebrate's principal means of delivering oxygen (O2) to the body tissues—via blood flow through the circulatory system. RBCs take up oxygen in the lungs, or in fish the gills, and release it into tissues while squeezing through the body's capillaries. The cytoplasm of a red blood cell is rich in hemoglobin, an iron-containing biomolecule that can bind oxygen and is responsible for the red color of the cells and the blood. Each human red blood cell contains approximately 270 million hemoglobin molecules. The cell membrane is composed of proteins and lipids, and this structure provides properties essential for physiologi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
SMAD (protein)
Smads (or SMADs) comprise a family of structurally similar proteins that are the main signal transducers for receptors of the transforming growth factor beta (TGF-B) superfamily, which are critically important for regulating cell development and growth. The abbreviation refers to the homologies to the ''Caenorhabditis elegans'' SMA ("small" worm phenotype) and MAD family ("Mothers Against Decapentaplegic") of genes in Drosophila. There are three distinct sub-types of Smads: receptor-regulated Smads ( R-Smads), common partner Smads (Co-Smads), and inhibitory Smads ( I-Smads). The eight members of the Smad family are divided among these three groups. Trimers of two receptor-regulated SMADs and one co-SMAD act as transcription factors that regulate the expression of certain genes. Sub-types The R-Smads consist of Smad1, Smad2, Smad3, Smad5 and Smad8/9, and are involved in direct signaling from the TGF-B receptor. Smad4 is the only known human Co-Smad, and has the role of partneri ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Ligands
In coordination chemistry, a ligand is an ion or molecule (functional group) that binds to a central metal atom to form a coordination complex. The bonding with the metal generally involves formal donation of one or more of the ligand's electron pairs, often through Lewis bases. The nature of metal–ligand bonding can range from covalent to ionic. Furthermore, the metal–ligand bond order can range from one to three. Ligands are viewed as Lewis bases, although rare cases are known to involve Lewis acidic "ligands". Metals and metalloids are bound to ligands in almost all circumstances, although gaseous "naked" metal ions can be generated in a high vacuum. Ligands in a complex dictate the reactivity of the central atom, including ligand substitution rates, the reactivity of the ligands themselves, and redox. Ligand selection requires critical consideration in many practical areas, including bioinorganic and medicinal chemistry, homogeneous catalysis, and environmental chemis ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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