Cerezyme
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]   |
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Genzyme
Genzyme was an American biotechnology company based in Cambridge, Massachusetts. Since its acquisition in 2011, Genzyme (also known as Genzyme Transgenics Corp or GTC Biotherapeutics) has been a fully owned subsidiary of Sanofi. In 2010, Genzyme was the world’s third-largest biotechnology company, employing more than 11,000 people around the world. As a subsidiary of Sanofi, Genzyme has a presence in approximately 65 countries, including 17 manufacturing facilities and 9 genetic-testing laboratories. Its products are also sold in 90 countries. In 2007, Genzyme generated $3.8 billion in revenue with more than 25 products on the market. In 2006 and 2007, Genzyme was named one of ''Fortune'' magazine’s “100 Best Companies to Work for”. The company donated $83 million worth of products worldwide; in 2006, it made $11 million in cash donations. In 2005, Genzyme was awarded the National Medal of Technology, the highest level of honor awarded by the president of the United States ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Genzyme Corporation
Genzyme was an American biotechnology company based in Cambridge, Massachusetts. Since its acquisition in 2011, Genzyme (also known as Genzyme Transgenics Corp or GTC Biotherapeutics) has been a fully owned subsidiary of Sanofi. In 2010, Genzyme was the world’s third-largest biotechnology company, employing more than 11,000 people around the world. As a subsidiary of Sanofi, Genzyme has a presence in approximately 65 countries, including 17 manufacturing facilities and 9 genetic-testing laboratories. Its products are also sold in 90 countries. In 2007, Genzyme generated $3.8 billion in revenue with more than 25 products on the market. In 2006 and 2007, Genzyme was named one of ''Fortune'' magazine’s “100 Best Companies to Work for”. The company donated $83 million worth of products worldwide; in 2006, it made $11 million in cash donations. In 2005, Genzyme was awarded the National Medal of Technology, the highest level of honor awarded by the president of the United States ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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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]   |
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Vesivirus
''Vesivirus'' is a genus of viruses, in the family '' Caliciviridae''. Swine, sea mammals, and felines serve as natural hosts. There are two species in this genus. Diseases associated with this genus include: respiratory disease, ''Feline calicivirus'' (FCV); conjunctivitis, and respiratory disease. Taxonomy The genus contains the following two species: *''Feline calicivirus'' *'' Vesicular exanthema of swine virus'' Other, unofficial Vesi-like viruses include canine calicivirus, San Miguel sealion virus, vesivirus Cro1 and walrus calicivirus. Virion properties Morphology Virions consist of a capsid. Virus capsid is non-enveloped, and are round with icosahedral symmetry and triangulation number T=3. The isometric capsid has a diameter of 35–39 nm. Empty virions have diameter of 23 nm, with triangulation number T=1. The capsid surface structure reveals a regular pattern with distinctive features, including 32 cup-shaped depressions. The capsomer arrangement is clea ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Glucocerebrosidase
β-Glucocerebrosidase (also called acid β-glucosidase, D-glucosyl-N-acylsphingosine glucohydrolase, or GCase) is an enzyme with glucosylceramidase activity () that is needed to cleave, by hydrolysis, the beta-glycosidic linkage of the chemical glucocerebroside, an intermediate in glycolipid metabolism that is abundant in cell membranes (particularly skin cells). It is localized in the lysosome, where it remains associated with the lysosomal membrane. β-Glucocerebrosidase is 497 amino acids in length and has a molecular weight of 59,700 Daltons. Structure β-Glucocerebrosidase is a member of the glycoside hydrolase family 30 and consists of three distinct domains (I-III). File:Structure of human beta-glucocerebrosidase @.png, Three-dimensional PyMol rendering of glucocerebrosidase with three domains highlighted. File:Glucocerebrosidase active site.png, Three-dimensional PyMol rendering of glucocerebrosidase with catalytic residues highlighted. Domain I (residues 1–27 and 38 ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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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]   |
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Velaglucerase Alfa
Velaglucerase alfa, sold under the brand name Vpriv and manufactured by Shire plc, is a hydrolytic lysosomal glucocerebroside-specific enzyme, which is a recombinant form of glucocerebrosidase indicated as a long-term enzyme replacement therapy for those with Gaucher disease Type 1. It has an identical amino acid sequence to the naturally occurring enzyme. It was approved for use by the U.S. Food and Drug Administration (FDA) on February 26, 2010. The most common side effects include abdominal (belly) pain, headache, dizziness, bone pain, arthralgia (joint pain), back pain, infusion-related reactions, asthenia (weakness) or fatigue (tiredness), and pyrexia (fever) or increased body temperature. Velaglucerase alfa was approved for medical use in the European Union in August 2010. Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. Medical uses Velaglucerase alfa is indicated for long-term enzyme ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Rashes
A rash is a change of the human skin which affects its color, appearance, or texture. A rash may be localized in one part of the body, or affect all the skin. Rashes may cause the skin to change color, itch, become warm, bumpy, chapped, dry, cracked or blistered, swell, and may be painful. The causes, and therefore treatments for rashes, vary widely. Diagnosis must take into account such things as the appearance of the rash, other symptoms, what the patient may have been exposed to, occupation, and occurrence in family members. The diagnosis may confirm any number of conditions. The presence of a rash may aid diagnosis; associated signs and symptoms are diagnostic of certain diseases. For example, the rash in measles is an erythematous, morbilliform, maculopapular rash that begins a few days after the fever starts. It classically starts at the head, and spreads downwards. Differential diagnosis Common causes of rashes include: * Food allergy * Medication side effects * Anxiety ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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Orphan Drugs
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]   |
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Drugs Acting On The Gastrointestinal System And Metabolism
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]   |
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Taliglucerase Alfa
Taliglucerase alfa, sold under the brand name Elelyso among others, is a biopharmaceutical medication developed by Protalix and Pfizer. The drug, a recombinant glucocerebrosidase used to treat Gaucher's disease, is the first plant-made pharmaceutical to win approval by the U.S. Food and Drug Administration (FDA). Each vial has 200 units of taliglucerase alfa. Approval history The U.S. FDA New Drug Application (NDA) was granted approval in May 2012, for use in adults. The U.S. FDA Supplemental New Drug Application (sNDA) for pediatric use was granted approved in August 2014. In Israel, the Israeli Ministry of Health granted approval in September 2012. In Brazil, the Brazilian Health Surveillance Agency (ANVISA) granted approval in March 2013. In Canada, Health Canada issued a Notice of Compliance in May 2014, for both adults and pediatric patients. Taliglucerase alfa is made by the Israeli biotherapeutics company Protalix and sold by the American pharmaceutical company Pfizer ... [...More Info...]       [...Related Items...]     OR:     [Wikipedia]   [Google]   [Baidu]   |
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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]   |