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Prosensa
Prosensa was a biotechnology company engaged in the discovery, development and commercialization of RNA-modulating therapeutics. The company targets genetic disorders with a large unmet medical need, with a primary focus on neuromuscular and neurodegenerative disorders such as Duchenne muscular dystrophy (DMD), myotonic dystrophy, and Huntington's disease. Prosensa was acquired by BioMarin. History up to the sale Prosensa commenced operations in 2002 and is located in Leiden, The Netherlands. The company works closely together with academia, patient groups and experts worldwide. Prosensa entered into an exclusive licensing agreement in 2003 with Leiden University Medical Center (LUMC) for their proprietary RNA modulation exon-skipping technology to develop treatments for DMD, other neuromuscular disorders and indications outside the field of neuromuscular disorders. Prosensa's portfolio of clinical and pre-clinical RNA-based drug candidates is currently focused on the treatment o ...
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Drisapersen
Drisapersen (also known as Kyndrisa, PRO051 and GSK2402968) is an experimental drug that was under development by BioMarin, after acquisition of Prosensa, for the treatment of Duchenne muscular dystrophy. The drug is a 2'-O-methyl phosphorothioate oligonucleotide that alters the splicing of the dystrophin RNA transcript, eliminating exon 51 from the mature dystrophin mRNA. Mechanism of action Duchenne muscular dystrophy (DMD) is caused when a mutation in the dystrophin gene changes the ribonucleic acid, RNA so that it no longer codes for functional dystrophin protein. This usually happens due to a mutation that alters the reading frame of the RNA Upstream and downstream (DNA), downstream of the mutation, so-called frameshift mutation. If an exon with an appropriate number of nucleobase, bases lies near the mutation, removing that exon can correct the downstream reading frame, restoring the production of partially functional dystrophin. This is the general strategy used in the des ...
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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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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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PRO044
Pro is an abbreviation meaning "professional". Pro, PRO or variants thereof might also refer to: People * Miguel Pro (1891–1927), Mexican priest * Pro Hart (1928–2006), Australian painter * Mlungisi Mdluli (born 1980), South African retired footballer * Derek Minor (born 1984), American Christian rapper, formerly known as PRo * Mike Awesome (1965–2007), a.k.a. The Pro, American wrestler Michael Lee Alfonso * Pro Wells, American football player Occupations * Prostitute, slang abbreviation * Public relations officer Linguistics * PRO (linguistics) ("big PRO") * pro (linguistics) ("little pro") Political parties * ' (Republican Proposal), Argentina * ', ( Progressive Party), Chile * ' (Law, Respect, Expertise), Czech Republic * ' ( Party for a Rule of Law Offensive), former German party * ' ( The Progressives), Latvia * ', 2020–2023 name of the Modern Democratic Party in Moldova * ' (PRO Romania) * ' (Proposition), Ukraine * ' (Pro-movement), Germany Organizations ...
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2015 Mergers And Acquisitions
Fifteen or 15 may refer to: *15 (number) *one of the years 15 BC, AD 15, 1915, 2015 Music * Fifteen (band), a punk rock band Albums * ''15'' (Buckcherry album), 2005 * ''15'' (Ani Lorak album), 2007 * ''15'' (Phatfish album), 2008 * ''15'' (Tuki album), 2025 * ''15'' (mixtape), a 2018 mixtape by Bhad Bhabie * ''Fifteen'' (Green River Ordinance album), 2016 * ''Fifteen'' (The Wailin' Jennys album), 2017 * ''Fifteen'', a 2012 album by Colin James Songs * "Fifteen" (song), a 2008 song by Taylor Swift *"Fifteen", a song by Harry Belafonte from the album '' Love Is a Gentle Thing'' *"15", a song by Rilo Kiley from the album ''Under the Blacklight'' *"15", a song by Marilyn Manson from the album ''The High End of Low'' Other media * ''15'' (film), a 2003 Singaporean film * ''Fifteen'' (TV series), international release name of ''Hillside'', a Canadian-American teen drama * "Fifteen" (''Runaways''), an episode of ''Runaways'' *Fifteen (novel), a 1956 juvenile fic ...
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Dutch Brands
Dutch or Nederlands commonly refers to: * Something of, from, or related to the Netherlands ** Dutch people as an ethnic group () ** Dutch nationality law, history and regulations of Dutch citizenship () ** Dutch language () * In specific terms, it reflects the Kingdom of the Netherlands ** Dutch Caribbean ** Netherlands Antilles Dutch may also refer to: Places * Dutch, West Virginia, a community in the United States * Pennsylvania Dutch Country People Ethnic groups * Pennsylvania Dutch, a group of early German immigrants to Pennsylvania Specific people * Dutch (nickname), a list of people * Johnny Dutch (born 1989), American hurdler and field athlete * Dutch Schultz (1902–1935), American mobster born Arthur Simon Flegenheimer * Dutch Mantel, ring name of American retired professional wrestler Wayne Maurice Keown (born 1949) * Dutch Savage, ring name of professional wrestler and promoter Frank Stewart (1935–2013) Arts, entertainment, and media Fictional charact ...
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Biotechnology Companies Disestablished In 2014
Biotechnology is a multidisciplinary field that involves the integration of natural sciences and engineering sciences in order to achieve the application of organisms and parts thereof for products and services. Specialists in the field are known as biotechnologists. The term ''biotechnology'' was first used by Károly Ereky in 1919 to refer to the production of products from raw materials with the aid of living organisms. The core principle of biotechnology involves harnessing biological systems and organisms, such as bacteria, yeast, and plants, to perform specific tasks or produce valuable substances. Biotechnology had a significant impact on many areas of society, from medicine to agriculture to environmental science. One of the key techniques used in biotechnology is genetic engineering, which allows scientists to modify the genetic makeup of organisms to achieve desired outcomes. This can involve inserting genes from one organism into another, and consequently, create ne ...
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Biotechnology Companies Established In 2002
Biotechnology is a multidisciplinary field that involves the integration of natural sciences and engineering sciences in order to achieve the application of organisms and parts thereof for products and services. Specialists in the field are known as biotechnologists. The term ''biotechnology'' was first used by Károly Ereky in 1919 to refer to the production of products from raw materials with the aid of living organisms. The core principle of biotechnology involves harnessing biological systems and organisms, such as bacteria, yeast, and plants, to perform specific tasks or produce valuable substances. Biotechnology had a significant impact on many areas of society, from medicine to agriculture to environmental science. One of the key techniques used in biotechnology is genetic engineering, which allows scientists to modify the genetic makeup of organisms to achieve desired outcomes. This can involve inserting genes from one organism into another, and consequently, create n ...
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Companies Formerly Listed On The Nasdaq
A company, abbreviated as co., is a legal entity representing an association of legal people, whether natural, juridical or a mixture of both, with a specific objective. Company members share a common purpose and unite to achieve specific, declared goals. Over time, companies have evolved to have the following features: "separate legal personality, limited liability, transferable shares, investor ownership, and a managerial hierarchy". The company, as an entity, was created by the state which granted the privilege of incorporation. Companies take various forms, such as: * voluntary associations, which may include nonprofit organizations * business entities, whose aim is to generate sales, revenue, and profit * financial entities and banks * programs or educational institutions A company can be created as a legal person so that the company itself has limited liability as members perform or fail to discharge their duties according to the publicly declared incorporation ...
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Biotechnology Companies Of The Netherlands
Biotechnology is a multidisciplinary field that involves the integration of natural sciences and engineering sciences in order to achieve the application of organisms and parts thereof for products and services. Specialists in the field are known as biotechnologists. The term ''biotechnology'' was first used by Károly Ereky in 1919 to refer to the production of products from raw materials with the aid of living organisms. The core principle of biotechnology involves harnessing biological systems and organisms, such as bacteria, yeast, and plants, to perform specific tasks or produce valuable substances. Biotechnology had a significant impact on many areas of society, from medicine to agriculture to environmental science. One of the key techniques used in biotechnology is genetic engineering, which allows scientists to modify the genetic makeup of organisms to achieve desired outcomes. This can involve inserting genes from one organism into another, and consequently, create n ...
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De Volkskrant
''De Volkskrant'' (; ), stylized as de Volkskrant, is a Dutch daily morning newspaper. Founded in 1919, it has a nationwide circulation of about 250,000. Formerly a leading centre-left Catholic broadsheet, ''de Volkskrant'' today is a medium-sized centrist compact. Pieter Klok is the current editor-in-chief. History and profile ''De Volkskrant'' was founded in 1919 and has been a daily morning newspaper since 1921. Originally ''de Volkskrant'' was a Roman Catholic newspaper closely linked to the Catholic People's Party and the Catholic pillar. The paper temporarily ceased publication in 1941. On its re-founding in 1945, its office moved from Den Bosch to Amsterdam Amsterdam ( , ; ; ) is the capital of the Netherlands, capital and Municipalities of the Netherlands, largest city of the Kingdom of the Netherlands. It has a population of 933,680 in June 2024 within the city proper, 1,457,018 in the City Re .... It became a left-wing newspaper in the 1960s, but began s ...
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Dystrophin
Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane. This complex is variously known as the costamere or the dystrophin-associated protein complex (DAPC). Many muscle proteins, such as α-dystrobrevin, syncoilin, synemin, sarcoglycan, dystroglycan, and sarcospan, colocalize with dystrophin at the costamere. It has a molecular weight of 427 kDa. Dystrophin is coded for by the ''DMD'' gene – the largest known human gene, covering 2.4 megabases (0.08% of the human genome) at Locus (genetics), locus X chromosome, Xp21. The primary transcript in muscle measures about 2,100 kilobases and takes 16 hours to transcribe; the Mature messenger RNA, mature mRNA measures 14.0 kilobases. The 79-exon muscle transcript codes for a protein of 3685 amino acid residues. Spontaneous or inherited mutations in the dystrophin gene can cause different form ...
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