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Synageva
Synageva BioPharma Corp. was a publicly listed biopharmaceutical company () headquartered in Lexington, Massachusetts dedicated to discovering, developing and delivering medicines for patients with rare diseases and high unmet medical needs. The company had manufacturing and laboratory locations in Lexington and Holden, Massachusetts, Bogart and Athens Georgia, as well as offices in a variety of locations around the world. History Synageva was formed when Sanj K. Patel (formerly an executive at Genzyme Corporation, Cambridge, Massachusetts) was approached in early 2008 by Baker Bros. Investments to be the CEO of privately-held Avigenics, Inc. in Athens, Georgia, a company initially formed by Dr. Robert Ivarie of the University of Georgia. Upon joining AviGenics in June 2008, Mr. Patel created the vision and strategy for Synageva and changed the company's direction to focus on products for the treatment of devastating, rare diseases. This represented a substantial shift in the ori ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Alexion Pharmaceuticals
Alexion Pharmaceuticals is an American pharmaceutical company headquartered in Boston, Massachusetts that specializes in orphan drugs to treat rare diseases. It became an independent subsidiary of AstraZeneca in 2021. Its products include eculizumab (Soliris) with $4.064 billion in 2020 revenues and ravulizumab (Ultomiris) with $1.076 billion in 2020 revenues, both used to treat the rare disorders of atypical hemolytic uremic syndrome (aHUS) and paroxysmal nocturnal hemoglobinuria (PNH); asfotase alfa (Strensiq) with $731 million in 2020 revenues, used to treat hypophosphatasia; sebelipase alfa (Kanuma) with $117 million in 2020 revenues, used to treat lysosomal acid lipase deficiency, and andexanet alfa (Andexxa) with $78 million in 2020 revenues, used to stop life threatening or uncontrollable bleeding in people who are taking rivaroxaban or apixaban. With costs that can reach as much as $2 million per year, the drugs manufactured by Alexion are some of the most expensive dru ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Sebelipase Alfa
Sebelipase alfa, sold under the brand name Kanuma, is a recombinant form of the enzyme lysosomal acid lipase (LAL) that is used as a medication for the treatment of lysosomal acid lipase deficiency (LAL-D). Text was copied from this source which is © European Medicines Agency. Reproduction is authorized provided the source is acknowledged. It is administered via intraveneous infusion. It was approved for medical use in the European Union and in the United States in 2015. Medical uses Sebelipase alfa is indicated for long-term enzyme replacement therapy (ERT) in people of all ages with lysosomal acid lipase (LAL) deficiency. History Sebelipase was developed by Synageva that became part of Alexion Pharmaceuticals in 2015. For its production, chickens are genetically modified to produce the recombinant form of LAL (rhLAL) in their egg white. After extraction and purification it becomes available as the medication. On 8 December 2015 the FDA announced that its approval came ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Lexington, Massachusetts
Lexington is a suburban town in Middlesex County, Massachusetts, United States. It is 10 miles (16 km) from Downtown Boston. The population was 34,454 as of the 2020 census. The area was originally inhabited by Native Americans, and was first settled by Europeans in 1641 as a farming community. Lexington is well known as the site of the first shots of the American Revolutionary War, in the Battle of Lexington on April 19, 1775, where the " Shot heard 'round the world" took place. It is home to Minute Man National Historical Park. History Indigenous history Native Americans inhabited the area that would become Lexington for thousands of years prior to European colonization of the Americas, as attested by a woodland era archaeological site near Loring Hill south of the town center. At the time of European contact, the area may have been a border region between Naumkeag or Pawtucket to the northeast, Massachusett to the south, and Nipmuc to the west, though the land was ev ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Biopharmaceutical
A biopharmaceutical, also known as a biological medical product, or biologic, is any pharmaceutical drug product manufactured in, extracted from, or semisynthesized from biological sources. Different from totally synthesized pharmaceuticals, they include vaccines, whole blood, blood components, allergenics, somatic cells, gene therapies, tissues, recombinant therapeutic protein, and living medicines used in cell therapy. Biologics can be composed of sugars, proteins, nucleic acids, or complex combinations of these substances, or may be living cells or tissues. They (or their precursors or components) are isolated from living sources—human, animal, plant, fungal, or microbial. They can be used in both human and animal medicine. Terminology surrounding biopharmaceuticals varies between groups and entities, with different terms referring to different subsets of therapeutics within the general biopharmaceutical category. Some regulatory agencies use the terms ''biological ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Chief Executive Officer
A chief executive officer (CEO), also known as a central executive officer (CEO), chief administrator officer (CAO) or just chief executive (CE), is one of a number of corporate executives charged with the management of an organization especially an independent legal entity such as a company or nonprofit institution. CEOs find roles in a range of organizations, including public and private corporations, non-profit organizations and even some government organizations (notably state-owned enterprises). The CEO of a corporation or company typically reports to the board of directors and is charged with maximizing the value of the business, which may include maximizing the share price, market share, revenues or another element. In the non-profit and government sector, CEOs typically aim at achieving outcomes related to the organization's mission, usually provided by legislation. CEOs are also frequently assigned the role of main manager of the organization and the highest-ranking offic ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Lysosomal Acid Lipase Deficiency
Lysosomal acid lipase deficiency (LAL deficiency or LAL-D) is an autosomal recessive inborn error of metabolism that results in the body not producing enough active lysosomal acid lipase (LAL) enzyme. This enzyme plays an important role in breaking down fatty material (cholesteryl esters and triglycerides) in the body. Infants, children and adults that have LAL deficiency experience a range of serious health problems. The lack of the LAL enzyme can lead to a build-up of fatty material in a number of body organs including the liver, spleen, gut, in the wall of blood vessels and other important organs. Very low levels of the LAL enzyme lead to LAL deficiency. LAL deficiency typically affects infants in the first year of life. The accumulation of fat in the walls of the gut in early onset disease leads to serious digestive problems including malabsorption, a condition in which the gut fails to absorb nutrients and calories from food. Because of these digestive complications, affecte ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Hunter Syndrome
Hunter syndrome, or mucopolysaccharidosis type II (MPS II), is a rare genetic disorder in which large sugar molecules called glycosaminoglycans (or GAGs or mucopolysaccharides) build up in body tissues. It is a form of lysosomal storage disease. Hunter syndrome is caused by a deficiency of the lysosomal enzyme iduronate-2-sulfatase (I2S). The lack of this enzyme causes heparan sulfate and dermatan sulfate to accumulate in all body tissues. Hunter syndrome is the only MPS syndrome to exhibit X-linked recessive inheritance. The symptoms of Hunter syndrome are comparable to those of MPS I. It causes abnormalities in many organs, including the skeleton, heart, and respiratory system. In severe cases, this leads to death during the teenaged years. Unlike MPS I, corneal clouding is not associated with this disease. Signs and symptoms Hunter syndrome may present with a wide variety of phenotypes. It has traditionally been categorized as either "mild" or "severe" depending on the pre ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Fabry Disease
Fabry disease, also known as Anderson–Fabry disease, is a rare genetic disease that can affect many parts of the body, including the kidneys, heart, and skin. Fabry disease is one of a group of conditions known as lysosomal storage diseases. The genetic mutation that causes Fabry disease interferes with the function of an enzyme that processes biomolecules known as sphingolipids, leading to these substances building up in the walls of blood vessels and other organs. It is inherited in an X-linked manner. Fabry disease is sometimes diagnosed using a blood test that measures the activity of the affected enzyme called alpha-galactosidase, but genetic testing is also sometimes used, particularly in females. The treatment for Fabry disease varies depending on the organs affected by the condition, and the underlying cause can be addressed by replacing the enzyme that is lacking. The first descriptions of the condition were made simultaneously by dermatologist Johannes Fabry and ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Pompe Disease
Glycogen storage disease type II, also called Pompe disease, is an autosomal recessive metabolic disorder which damages muscle and nerve cells throughout the body. It is caused by an accumulation of glycogen in the lysosome due to deficiency of the lysosomal acid alpha-glucosidase enzyme. It is the only glycogen storage disease with a defect in lysosomal metabolism, and the first glycogen storage disease to be identified, in 1932 by the Dutch pathologist J. C. Pompe. The build-up of glycogen causes progressive muscle weakness (myopathy) throughout the body and affects various body tissues, particularly in the heart, skeletal muscles, liver and the nervous system. Signs and symptoms Newborn The infantile form usually comes to medical attention within the first few months of life. The usual presenting features are cardiomegaly (92%), hypotonia (88%), cardiomyopathy (88%), respiratory distress (78%), muscle weakness (63%), feeding difficulties (57%) and failure to thrive (50%). ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
American Companies Established In 1996
American(s) may refer to: * American, something of, from, or related to the United States of America, commonly known as the "United States" or "America" ** Americans, citizens and nationals of the United States of America ** American ancestry, people who self-identify their ancestry as "American" ** American English, the set of varieties of the English language native to the United States ** Native Americans in the United States, indigenous peoples of the United States * American, something of, from, or related to the Americas, also known as "America" ** Indigenous peoples of the Americas * American (word), for analysis and history of the meanings in various contexts Organizations * American Airlines, U.S.-based airline headquartered in Fort Worth, Texas * American Athletic Conference, an American college athletic conference * American Recordings (record label), a record label previously known as Def American * American University, in Washington, D.C. Sports teams Soccer * ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Health Care Companies Based In Massachusetts
Health, according to the World Health Organization, is "a state of complete physical, mental and social well-being and not merely the absence of disease and infirmity".World Health Organization. (2006)''Constitution of the World Health Organization''– ''Basic Documents'', Forty-fifth edition, Supplement, October 2006. A variety of definitions have been used for different purposes over time. Health can be promoted by encouraging healthful activities, such as regular physical exercise and adequate sleep, and by reducing or avoiding unhealthful activities or situations, such as smoking or excessive stress. Some factors affecting health are due to individual choices, such as whether to engage in a high-risk behavior, while others are due to structural causes, such as whether the society is arranged in a way that makes it easier or harder for people to get necessary healthcare services. Still, other factors are beyond both individual and group choices, such as genetic disorders. ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Companies Listed On The Nasdaq
A company, abbreviated as co., is a legal entity representing an association of people, whether natural, legal or a mixture of both, with a specific objective. Company members share a common purpose and unite to achieve specific, declared goals. Companies take various forms, such as: * voluntary associations, which may include nonprofit organizations * business entities, whose aim is generating 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 duty according to the publicly declared incorporation, or published policy. When a company closes, it may need to be liquidated to avoid further legal obligations. Companies may associate and collectively register themselves as new companies; the resulting entities are often known as corporate groups. Meanings and definitions A company can be defined as an "artificial per ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
