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Amicus Therapeutics
Amicus Therapeutics is a public American biopharmaceutical company based in Philadelphia, PA. The company went public in 2007 under the NASDAQ trading symbol FOLD. This followed a 2006 planned offering and subsequent withdrawal, which would have established the trading symbol as AMTX Prior to their IPO, Amicus was funded by a variety of venture capital firms including Radius Ventures, Canaan Partners and New Enterprise Associates. History The therapeutic focus of Amicus is on rare and orphan diseases, particularly disorders collectively called lysosomal storage disorders. The company has focused on pharmacological chaperones and enzyme replacement therapy. In 2008, the company expanded from its single site in New Jersey to a second research site in San Diego. In late 2009, the company faced a major financial setback with the termination of a multi-year collaboration agreement with Shire for its lead compound, migalastat, and two other products. Amicus cut 20% of its workforc ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Public Company
A public company is a company whose ownership is organized via shares of stock which are intended to be freely traded on a stock exchange or in over-the-counter markets. A public (publicly traded) company can be listed on a stock exchange (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 therefore 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 Kingdom it is usually a public limited company (plc), i ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Seeking Alpha
Seeking Alpha is a crowd-sourced content service for financial markets. Articles and research covers a broad range of stocks, asset classes, exchange-traded funds (ETFs), and investment strategies. Unlike other equity research platforms, insight is provided by contributors including a base of investors and industry experts (buy side) rather than sell side. Seeking Alpha was founded in 2004 by former Wall Street analyst David Jackson. In 2011 the company stated it had distribution partnerships with MSN Money, CNBC, Yahoo! Finance, MarketWatch, NASDAQ and TheStreet. Yahoo Finance ended its relationship with Seeking Alpha on July 28, 2014. As of January 2021, the firm self-reported that it had 10 million registered users and attracts over 17 million unique viewers every month. Its average visit duration was 4× more than ''The Economist'', ''Barron's'' or the ''Wall Street Journal.'' Contributors The site's content is primarily generated by independent contributors. As of January ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Scioderm
Scioderm, acquired by Amicus Therapeutics in 2015, was a rare disease company focused on developing a treatment for Epidermolysis Bullosa (EB), a rare genetic disease characterized by extremely fragile skin and recurrent blister formation. There are currently no approved therapies for EB. Scioderm was developing a topical treatment known as SD-101, or Zorblisa, aimed at triggering wound reduction and closure, and a reduction in body surface area coverage of blisters and lesions. Epidermolysis Bullosa, sometimes referred to as "Butterfly Skin", is a rare genetic connective tissue disorder that, in all forms, results in extremely fragile skin that blisters or tears at the slightest friction or trauma. EB typically manifests at birth or early childhood. According to the Dystrophic Epidermolysis Bullosa Research Association of America (DEBRA), an estimated 1 out of every 20,000 live births are affected with some type of EB and the disorder occurs in every racial and ethnic group ... [...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] |
GlaxoSmithKline
GSK plc, formerly GlaxoSmithKline plc, is a British multinational pharmaceutical and biotechnology company with global headquarters in London, England. Established in 2000 by a merger of Glaxo Wellcome and SmithKline Beecham. GSK is the tenth largest pharmaceutical company and #294 on the 2022 ''Fortune'' Global 500, ranked behind other pharmaceutical companies China Resources, Sinopharm, Johnson & Johnson, Pfizer, Roche, AbbVie, Novartis, Bayer, and Merck. The company has a primary listing on the London Stock Exchange and is a constituent of the FTSE 100 Index. , it had a market capitalisation of £70 billion, the eighth largest on the London Stock Exchange. It has a secondary listing on the New York Stock Exchange. The company developed the first malaria vaccine, RTS,S, which it said in 2014 it would make available for five percent above cost. Legacy products developed at GSK include several listed in the World Health Organization's List of Essential Medicines, such ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Migalastat
Migalastat, sold under the brand name Galafold, is a medication for the treatment of Fabry disease, a rare genetic disorder. It was developed by Amicus Therapeutics. The US Food and Drug Administration (FDA) granted it orphan drug status in 2004, and the European Commission followed in 2006. The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) granted the drug a marketing approval under the name Galafold in May 2016. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses Migalastat is used for the long-term treatment of Fabry disease in adults and adolescents aged 16 or older with an amenable mutation of the enzyme alpha-galactosidase A (α-GalA). An "amenable" mutation is one that leads to misfolding of the enzyme, but otherwise would not significantly impair its function. Based on an ''in vitro'' test, Amicus Therapeutics has published a list of 269 amenable and nearly 600 non-amenable mutation ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Shire (pharmaceutical Company)
Shire plc was a UK-founded Jersey-registered specialty biopharmaceutical company. Originating in the United Kingdom with an operational base in the United States, its brands and products included Vyvanse, Lialda, and Adderall XR. Shire was acquired by Takeda Pharmaceutical Company on 8 January 2019. Shire was a global biotechnology company focused on serving people with rare diseases and other highly specialized conditions. The company's products were available in more than 100 countries across core therapeutic areas including Hematology, Immunology, Neuroscience, Lysosomal storage disorders, Lysosomal Storage Disorders, Gastrointestinal disease, Gastrointestinal / Internal medicine, Internal Medicine / Endocrine and Hereditary Angioedema; a growing franchise in Oncology; and an emerging, innovative pipeline in Ophthalmics. The original corporate headquarters was located in Basingstoke, Hampshire, England. Main offices are located in Dublin, Ireland, the United States in Cambr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
HighBeam Research
HighBeam Research was a paid search engine and full text online archive owned by Gale, a subsidiary of Cengage, for thousands of newspapers, magazines, academic journals, newswires, trade magazines, and encyclopedias in English. It was headquartered in Chicago, Illinois. In late 2018, the archive was shut down. History The company was established in August 2002 after Patrick Spain, who had just sold Hoover's, which he had co-founded, bought eLibrary and Encyclopedia.com from Tucows. The new company was called Alacritude, LLC (a combination of Alacrity and Attitude). ELibrary had a library of 1,200 newspaper, magazine and radio/TV transcript archives that were generally not freely available. Original investors included Prism Opportunity Fund of Chicago and 1 to 1 Ventures of Stamford, Connecticut. Spain stated, "There was a glaring gap between free search like Google and high-end offerings like LexisNexis and Factiva." Later in 2002, it bought Researchville.com. By 2003, it ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Biotech Week
''Biotech Week'' is a weekly biotechnology and pharmaceutical trade magazine. The magazine was established in 1999. It is published by NewsRX. The magazine covers a broad range of medical issues and financial related information about companies in these industries. The magazine is headquartered in Atlanta, Georgia Atlanta ( ) is the capital and most populous city of the U.S. state of Georgia. It is the seat of Fulton County, the most populous county in Georgia, but its territory falls in both Fulton and DeKalb counties. With a population of 498,7 .... References External links * * 1999 establishments in Georgia (U.S. state) English-language magazines Magazines established in 1999 Magazines published in Atlanta Medical magazines Professional and trade magazines Science and technology magazines published in the United States Weekly magazines published in the United States {{italic title ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
San Diego
San Diego ( , ; ) is a city on the Pacific Ocean coast of Southern California located immediately adjacent to the Mexico–United States border. With a 2020 population of 1,386,932, it is the List of United States cities by population, eighth most populous city in the United States and the county seat, seat of San Diego County, the List of the most populous counties in the United States, fifth most populous county in the United States, with 3,338,330 estimated residents as of 2019. The city is known for its mild year-round climate, natural deep-water harbor, extensive beaches and parks, long association with the United States Navy, and recent emergence as a healthcare and biotechnology development center. San Diego is the List of municipalities in California, second largest city in the U.S. state, state of California, after Los Angeles. Historically home to the Kumeyaay people, San Diego is frequently referred to as the "Birthplace of California", as it was the first site vi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Royal Society Of Chemistry
The Royal Society of Chemistry (RSC) is a learned society (professional association) in the United Kingdom with the goal of "advancing the chemistry, chemical sciences". It was formed in 1980 from the amalgamation of the Chemical Society, the Royal Institute of Chemistry, the Faraday Society, and the Society for Analytical Chemistry with a new Royal Charter and the dual role of learned society and professional body. At its inception, the Society had a combined membership of 34,000 in the UK and a further 8,000 abroad. The headquarters of the Society are at Burlington House, Piccadilly, London. It also has offices in Thomas Graham House in Cambridge (named after Thomas Graham (chemist), Thomas Graham, the first president of the Chemical Society) where ''RSC Publishing'' is based. The Society has offices in the United States, on the campuses of The University of Pennsylvania and Drexel University, at the University City Science Center in Philadelphia, Pennsylvania, in both Beijing a ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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 Univers ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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