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BioMarin Pharmaceutical
BioMarin Pharmaceutical Inc. is an American biotechnology company headquartered in San Rafael, California. It has offices and facilities in the United States, South America, Asia, and Europe. BioMarin's core business and research is in enzyme replacement therapies (ERTs). BioMarin was the first company to provide therapeutics for mucopolysaccharidosis type I (MPS I), by manufacturing laronidase ( Aldurazyme, commercialized by Genzyme Corporation). BioMarin was also the first company to provide therapeutics for phenylketonuria ( PKU). Over the years, BioMarin has been criticised for drug pricing and for specific instances of denying access to drugs in clinical trials. History BioMarin was founded in 1997 by Christopher Starr Ph.D. and Grant W. Denison Jr. with an investment of a $1.5 million from Glyko Biomedical and went public in 1999. Seed investors were amongst others MPM Bioventures, Grosvenor Fund and Florian Schönharting. Business development In 2002, BioMarin acqui ...
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Aldurazyme
Iduronidase (, ''L-iduronidase'', ''α-L-iduronidase'', ''laronidase''), sold as Aldurazyme, is an enzyme with the systematic name ''glycosaminoglycan α-L-iduronohydrolase''. It catalyses the hydrolysis of unsulfated α-L-iduronosidic linkages in dermatan sulfate.Aldurazyme (Laronidase)
BioMarin Pharmaceuticals Inc. FDA website. Retrieved 6 December 2015.
It is a glycoprotein enzyme found in the s of cells. It is involved in the degeneration of

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Public Company
A public company is a company whose ownership is organized via shares of share capital, stock which are intended to be freely traded on a stock exchange or in over-the-counter (finance), over-the-counter markets. A public (publicly traded) company can be listed on a stock exchange (listing (finance), listed company), which facilitates the trade of shares, or not (unlisted public company). In some jurisdictions, public companies over a certain size must be listed on an exchange. In most cases, public companies are ''private'' enterprises in the ''private'' sector, and "public" emphasizes their reporting and trading on the public markets. Public companies are formed within the legal systems of particular states and so have associations and formal designations, which are distinct and separate in the polity in which they reside. In the United States, for example, a public company is usually a type of corporation, though a corporation need not be a public company. In the United Kin ...
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Reuters
Reuters ( ) is a news agency owned by Thomson Reuters. It employs around 2,500 journalists and 600 photojournalists in about 200 locations worldwide writing in 16 languages. Reuters is one of the largest news agencies in the world. The agency was established in London in 1851 by Paul Reuter. The Thomson Corporation of Canada acquired the agency in a 2008 corporate merger, resulting in the formation of the Thomson Reuters Corporation. In December 2024, Reuters was ranked as the 27th most visited news site in the world, with over 105 million monthly readers. History 19th century Paul Julius Reuter worked at a book-publishing firm in Berlin and was involved in distributing radical pamphlets at the beginning of the Revolutions of 1848. These publications brought much attention to Reuter, who in 1850 developed a prototype news service in Aachen using homing pigeons and electric telegraphy from 1851 on, in order to transmit messages between Brussels and Aachen, in what today is Aa ...
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Lambert–Eaton Myasthenic Syndrome
Lambert–Eaton myasthenic syndrome (LEMS) is a rare autoimmune disorder characterized by muscle weakness of the limbs. It is also known as myasthenic syndrome, Eaton–Lambert syndrome, and when related to cancer, carcinomatous myopathy. Around 60% of those with LEMS have an underlying malignancy, most commonly small-cell lung cancer (SCLC); it is therefore regarded as a paraneoplastic syndrome (a condition that arises as a result of cancer elsewhere in the body). It is the result of antibodies against presynaptic voltage-gated calcium channels, and likely other nerve terminal proteins, in the neuromuscular junction (the connection between nerves and the muscle that they supply). The diagnosis is usually confirmed with electromyography and blood tests; these also distinguish it from myasthenia gravis, a related autoimmune neuromuscular disease. If the disease is associated with cancer, direct treatment of the cancer often relieves the symptoms of LEMS. Other treatments ...
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European Commission
The European Commission (EC) is the primary Executive (government), executive arm of the European Union (EU). It operates as a cabinet government, with a number of European Commissioner, members of the Commission (directorial system, informally known as "commissioners") corresponding to two thirds of the number of Member state of the European Union, member states, unless the European Council, acting unanimously, decides to alter this number. The current number of commissioners is 27, including the president. It includes an administrative body of about 32,000 European civil servants. The commission is divided into departments known as Directorate-General, Directorates-General (DGs) that can be likened to departments or Ministry (government department), ministries each headed by a director-general who is responsible to a commissioner. Currently, there is one member per European Union member state, member state, but members are bound by their oath of office to represent the genera ...
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Amifampridine Phosphate
Amifampridine phosphate is used as a drug, predominantly in the treatment of a number of rare muscle diseases. The free base form of the drug has been used to treat congenital myasthenic syndromes and approved by the FDA for Lambert–Eaton myasthenic syndrome (LEMS) through compassionate use programs since the 1990s and was recommended as a first line treatment for LEMS in 2006, using ''ad hoc'' forms of the drug, since there was no marketed form. Around 2000 doctors at Assistance Publique – Hôpitaux de Paris created a phosphate salt form, which was developed through a series of companies ending with BioMarin Pharmaceutical which obtained European approval in 2009 under the brand name Firdapse, and which licensed the US rights to Catalyst Pharmaceuticals in 2012. As of January 2017, Catalyst and another US company, Jacobus Pharmaceutical, which had been manufacturing and giving it away for free since the 1990s, were both seeking FDA approval for their iterations and mar ...
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Thomson Reuters
Thomson Reuters Corporation ( ) is a Canadian multinational corporation, multinational content-driven technology Conglomerate (company), conglomerate. The company was founded in Toronto, Ontario, Canada, and maintains its headquarters at 19 Duncan Street there. Thomson Reuters was created by the Thomson Corporation's purchase of the British company Reuters Group on 17 April 2008. It is majority-owned by the Woodbridge Company, a holding company for the Family tree of Thomson family, Thomson family of Canada. History Thomson Corporation The forerunner of the Thomson company was founded in 1934 by Roy Thomson, 1st Baron Thomson of Fleet, Roy Thomson in Ontario as the publisher of ''The Timmins Daily Press''. In 1953, Thomson acquired the ''The Scotsman, Scotsman'' newspaper and moved to Scotland the following year. He consolidated his media position in Scotland in 1957, when he won the government-granted monopoly, franchise for Scottish Television. In 1959, he bought the ...
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Genzyme
Genzyme (also known as Genzyme Transgenics Corp or GTC Biotherapeutics) was an American biotechnology company based in Cambridge, Massachusetts. From its acquisition in 2011 to 2022 Genzyme operated as 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 had a presence in approximately 65 countries, including 17 manufacturing facilities and 9 genetic-testing laboratories. Its products were 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 Unit ...
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Mucopolysaccharidosis
Mucopolysaccharidoses are a group of metabolic disorders caused by the absence or malfunctioning of lysosomal enzymes needed to break down molecules called glycosaminoglycans (GAGs). These long chains of sugar carbohydrates occur within the cells that help build bone, cartilage, tendons, corneas, skin and connective tissue. GAGs (formerly called mucopolysaccharides) are also found in the fluids that lubricate joints. Individuals with mucopolysaccharidosis either do not produce enough of one of the eleven enzymes required to break down these sugar chains into simpler molecules, or they produce enzymes that do not work properly. Over time, these GAGs collect in the cells, blood and connective tissues. The result is permanent, progressive cellular damage which affects appearance, physical abilities, organ and system functioning. The mucopolysaccharidoses are part of the lysosomal storage disease family, a group of genetic disorders that result when the lysosome organelle in anim ...
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Enzyme Replacement Therapy
Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme. ERT is available for some lysosomal storage diseases: Gaucher disease, Fabry disease, MPS I, MPS II (Hunter syndrome), MPS VI and Pompe disease. ERT does not correct the underlying genetic defect, but it increases the concentration of the enzyme that the patient is lacking. ERT has also been used to treat patients with severe combined immunodeficiency (SCID) resulting from an adenosine deaminase deficiency ( ADA-SCID). Other treatment options for patients with enzyme or protein deficiencies include substrate reduction therapy, gene therapy, and bone-marrow derived stem cell transplantation. History ERT was developed in 1964 by Christian de Duve and Roscoe Brady. Leading work was done on this subject at the Department of Physiology at the Un ...
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Europe
Europe is a continent located entirely in the Northern Hemisphere and mostly in the Eastern Hemisphere. It is bordered by the Arctic Ocean to the north, the Atlantic Ocean to the west, the Mediterranean Sea to the south, and Asia to the east. Europe shares the landmass of Eurasia with Asia, and of Afro-Eurasia with both Africa and Asia. Europe is commonly considered to be Boundaries between the continents#Asia and Europe, separated from Asia by the Drainage divide, watershed of the Ural Mountains, the Ural (river), Ural River, the Caspian Sea, the Greater Caucasus, the Black Sea, and the waterway of the Bosporus, Bosporus Strait. "Europe" (pp. 68–69); "Asia" (pp. 90–91): "A commonly accepted division between Asia and Europe ... is formed by the Ural Mountains, Ural River, Caspian Sea, Caucasus Mountains, and the Black Sea with its outlets, the Bosporus and Dardanelles." Europe covers approx. , or 2% of Earth#Surface, Earth's surface (6.8% of Earth's land area), making it ...
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