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Afegostat
Afegostat (International Nonproprietary Name, INN; also known as isofagomine; planned trade name Plicera) was an experimental drug for the treatment of certain forms of Gaucher's disease that was being developed by Amicus Therapeutics and Shire plc until a failed clinical trial in 2009 led to termination of its development. The substance was used in form of the tartrate. Mechanism of action β-Glucocerebrosidase, an enzyme needed for the metabolisation of glucocerebroside, is misfolded in individuals with Gaucher's disease due to various mutations, one of which is called N370S. Afegostat, an iminosugar, binds selectively to N370S glucocerebrosidase and restores its correct Protein structure, conformation and, consequently, enhances its activity about threefold. Invention and development Afegostat was invented by Mikael Bols and Troels Skrydstrup and was first prepared by Jespersen and Bols. Amicus Therapeutics licensed patents related to afegostat from Mt. Sinai School of Medici ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Imiglucerase
Imiglucerase is a medication used in the treatment of Gaucher's disease. It is a recombinant DNA-produced analogue of the human enzyme β-glucocerebrosidase. Cerezyme is a freeze-dried medicine containing imiglucerase, manufactured by Genzyme Corporation. It is given intravenously after reconstitution as a treatment for Type 1 and Type 3 Gaucher's disease. It is available in formulations containing 200 or 400 units per vial. The specific activity of highly purified human enzyme is 890,000 units/mg, meanwhile the enzyme activity produced by recombinant DNA technology is approximately 40 units/mg. A typical dose is 2.5U/kg every two weeks, up to a maximum of 60 U/kg once every two weeks, and safety has been established from ages 2 and up. It is one of more expensive medications, with an annual cost of $200,000 per person in the United States. Imiglucerase has been granted orphan drug status in the United States, Australia, and Japan. Cerezyme was one of the drugs manufactured at ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Novo Nordisk
Novo Nordisk A/S is a Danish multinational pharmaceutical company headquartered in Bagsværd, Denmark, with production facilities in nine countries, and affiliates or offices in five countries. Novo Nordisk is controlled by majority shareholder Novo Holdings A/S which holds approximately 25% of its shares and a supermajority (45%) of its voting shares. Novo Nordisk manufactures and markets pharmaceutical products and services specifically diabetes care medications and devices. Novo Nordisk is also involved with hemostasis management, growth hormone therapy and hormone replacement therapy. The company makes several drugs under various brand names, including Levemir, Tresiba, NovoLog, Novolin R, NovoSeven, NovoEight and Victoza. Novo Nordisk employs more than 48,000 people globally, and markets its products in 168 countries. The corporation was created in 1989 through a merger of two Danish companies which date back to the 1920s. The Novo Nordisk logo is the Apis bull, one of t ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Eliglustat
Eliglustat, sold under the brand name Cerdelga, is a medication used for the treatment of Gaucher's disease. It was discovered at the University of Michigan, developed by Genzyme, Genzyme Corp, and was approved by the FDA in August 2014. Commonly used as the tartrate salt (chemistry), salt, the compound is believed to work by inhibition of Ceramide glucosyltransferase, glucosylceramide synthase. According to an article in ''Journal of the American Medical Association'' the oral substrate reduction therapy resulted in "significant improvements in spleen volume, hemoglobin level, liver volume, and platelet count" in untreated adults with Gaucher disease Type 1. History Norman Radin began exploring the possibility of inhibiting the synthesis of lipid substrates involved in Gaucher's disease as early as 1982, and, in collaboration with the laboratory of Jim Shayman, found several candidate inhibitors in the mid-1990s. Genzyme initially rejected the candidates developed by Radin and Sha ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Miglustat
Miglustat, sold under the brand name Zavesca, is a medication used to treat type I Gaucher disease (GD1). It is also known as N-butyldeoxynojirimycin, and is a derivative of the anti-diabetic 1-deoxynojirimycin. It was developed by Oxford GlycoSciences and is marketed by Actelion. Miglustat has been approved in the EU, Japan, and Canada for treating progressive neurological complications in people with Niemann–Pick disease, type C (NPC). It was approved for medical use in the European Union in November 2002, and for medical use in the United States in July 2003. Medical uses Miglustat is indicated to treat adults with mild to moderate type I Gaucher disease for whom enzyme replacement therapy is unsuitable. Contraindications Miglustat is contraindicated for people with neurological conditions, kidney problems, women who are pregnant, and men and women planning to conceive a child. Adverse effects Serious side effects include pain, burning, numbness or tingling in the hands ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
US FDA
The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the 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 and over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, animal foods & feed and veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C), but 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 not directly related to food or drugs, but involves such things as regulating lasers, cellular phones, and condoms, as well as control of disease in contexts varying from h ... [...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] |
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] |
Mikael Bols
Mikael Bols (born July 28, 1961) is a synthetic organic chemistry, organic chemist who is mainly known for his work on carbohydrates and artificial enzymes. Early life Mikael Bols was born and grew up in Copenhagen, Denmark. He attended high school (Gentofte Statskole) from 1977 to 1980, and took a degree as chemical engineer at the Technical University of Denmark from 1980 to 1985. Professional career From 1985 to 1988, Bols did PhD with Professor Inge Lundt at the Technical University of Denmark. From 1988 to 1989, he did a post doc with professor Walter Szarek at Queen's University at Kingston, Queen's University before joining Leo Pharmaceuticals in 1989. From 1991 to 1995, he was Ass. Prof. at DTU only interrupted by a visit in the last 6 month of 1994 Gilbert Stork's group at Columbia University. From 1995 to 2000, he was Assoc. Prof. (Lektor) at University of Aarhus during which period he became Dr. Scient. (1997). From 1998 to 2000 and from 2004 to 2006, he was Head of the ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Gaucher's Disease
Gaucher's disease or Gaucher disease () (GD) is a genetic disorder A genetic disorder is a health problem caused by one or more abnormalities in the genome. It can be caused by a mutation in a single gene (monogenic) or multiple genes (polygenic) or by a chromosomal abnormality. Although polygenic disorders ... in which glucocerebroside (a sphingolipid, also known as glucosylceramide) accumulates in cells and certain organs. The disorder is characterized by bruising, fatigue (medical), fatigue, anemia, low blood platelet count and enlargement of the liver and spleen, and is caused by a hereditary deficiency of the enzyme glucocerebrosidase (also known as glucosylceramidase), which acts on glucocerebroside. When the enzyme is defective, glucocerebroside accumulates, particularly in white blood cells and especially in macrophages (mononuclear leukocytes, which is often a target for intracellular parasites). Glucocerebroside can collect in the spleen, liver, kidneys, Human lung, ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Protein Structure
Protein structure is the three-dimensional arrangement of atoms in an amino acid-chain molecule. Proteins are polymers specifically polypeptides formed from sequences of amino acids, the monomers of the polymer. A single amino acid monomer may also be called a ''residue'' indicating a repeating unit of a polymer. Proteins form by amino acids undergoing condensation reactions, in which the amino acids lose one water molecule per reaction in order to attach to one another with a peptide bond. By convention, a chain under 30 amino acids is often identified as a peptide, rather than a protein. To be able to perform their biological function, proteins fold into one or more specific spatial conformations driven by a number of non-covalent interactions such as hydrogen bonding, ionic interactions, Van der Waals forces, and hydrophobic packing. To understand the functions of proteins at a molecular level, it is often necessary to determine their three-dimensional structure. This is t ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Iminosugar
An iminosugar, also known as an iminosaccharide, is any analog of a sugar where a nitrogen atom has replaced the oxygen atom in the ring of the structure. Iminosugars are common components of plants and may be responsible for some of their medicinal properties. The first iminosugar to be isolated from a natural source, 1-deoxynojirimycin (DNJ), found in Mulberry, was reported in 1976, but few others were discovered until many years later. In terms of biochemical activity for medicinal applications, DNJ and 1,4-dideoxy-1,4-imino-D-arabinitol (DAB, another early example of this class of compounds) are alpha-glucosidase inhibitors and were shown to have anti-diabetic and anti-viral activity. DNJ was modified to produce two derivatives now used as medicines, ''N''-hydroxyethyl-DNJ (miglitol) for diabetes and ''N''-butyl-DNJ (miglustat) for Gaucher's disease. Anti-cancer and anti-viral activity was subsequently observed for swainsonine—a mannose analogue—and castanospermine—a glu ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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