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National Organization For Rare Disorders
The National Organization for Rare Disorders (NORD) is an American non-profit organization aiming to provide support for individuals with rare diseases by advocating and funding research, education, and networking among service providers. It was founded in 1983 by Abbey Meyers, along with individuals and rare diseases leaders of rare disease support groups, and it is a 501(c)3 tax exempt organization. History The organization grew out of an "informal coalition" of support groups and families called together in the late 1970s to advocate legislation supporting development of orphan drugs, or drugs for treating rare diseases. They succeeded in getting the United States Congress to pass the Orphan Drug Act (ODA) in early 1983. The initial coalition was led by Abbey Meyers, whose son had Tourette syndrome. Tourette syndrome was estimated by the National Institutes of Health to affect 100,000 people in the United States. Meyers' son was helped by an experimental drug that the manufa ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
501(c)(3)
A 501(c)(3) organization is a United States corporation, trust, unincorporated association or other type of organization exempt from federal income tax under section 501(c)(3) of Title 26 of the United States Code. It is one of the 29 types of 501(c) nonprofit organizations in the US. 501(c)(3) tax-exemptions apply to entities that are organized and operated exclusively for religious, charitable, scientific, literary or educational purposes, for testing for public safety, to foster national or international amateur sports competition, or for the prevention of cruelty to children or animals. 501(c)(3) exemption applies also for any non-incorporated community chest, fund, cooperating association or foundation organized and operated exclusively for those purposes.IR ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Internet
The Internet (or internet) is the global system of interconnected computer networks that uses the Internet protocol suite (TCP/IP) to communicate between networks and devices. It is a '' network of networks'' that consists of private, public, academic, business, and government networks of local to global scope, linked by a broad array of electronic, wireless, and optical networking technologies. The Internet carries a vast range of information resources and services, such as the inter-linked hypertext documents and applications of the World Wide Web (WWW), electronic mail, telephony, and file sharing. The origins of the Internet date back to the development of packet switching and research commissioned by the United States Department of Defense in the 1960s to enable time-sharing of computers. The primary precursor network, the ARPANET, initially served as a backbone for interconnection of regional academic and military networks in the 1970s to enable resource shari ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Alnylam Pharmaceuticals
Alnylam Pharmaceuticals, Inc. is an American biopharmaceutical company focused on the discovery, development and commercialization of RNA interference (RNAi) therapeutics for genetically defined diseases. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts. In 2016, ''Forbes'' included the company on its "100 Most Innovative Growth Companies" list. History The company is a spin-off from the Max Planck Institute for Biophysical Chemistry. In 2002, Alnylam was founded by scientists Phillip Sharp, Paul Schimmel, David Bartel, Thomas Tuschl, and Phillip Zamore, and by investors Christoph Westphal and John Kennedy Clarke; John Maraganore was the founding CEO. The company was named after Alnilam, a star in Orion’s belt. The spelling was modified to make it unique. In 2003, the firm merged with the German pharmaceutical company, Ribopharma AG. The newly formed company also received $24.6 million in funding from private-equity firms. On February 27, 2004 ... [...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] |
Agios Pharmaceuticals
Agios Pharmaceuticals Inc. is a public American pharmaceutical company pioneering therapies for genetically defined diseases, with a near-term focus on developing therapies for hemolytic anemias. The company was founded in 2008 (or 2007) by Lewis Cantley, Tak Mak and Craig Thompson. Agios is a Delaware corporation headquartered in Cambridge, Massachusetts. The company tendered an initial public offering in July 2013. History In 2012, Agios was named among the defendants in a lawsuit against one of its founders, Craig Thompson, alleging that Thompson used research illegally taken from the Abramson Family Cancer Research Institute in research at Agios. In May 2016, the company announced it would launch partnership with Celgene, developing metabolic immuno-oncology therapies and licensing AG-221 as well as AG-881 to Celgene, potentially garnering Agios $120 million in drug licensing payments. In April 2017, the company raised $250 million in a new stock offering in anticipation o ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Acadia Pharmaceuticals
Acadia Pharmaceuticals Inc. is a biopharmaceutical company headquartered in Sorrento Valley, San Diego, California. Product development Nuplazid, a drug developed by Acadia in the late 1990s, "was designed to stimulate a subset of the brain’s serotonin receptors, or the proteins that govern memory, cognition and learning." On April 29, 2016, the FDA approved Acadia's drug, Nuplazid, for the treatment of hallucinations and delusions associated with Parkinson's disease psychosis. Nuplazid is the trade name for Acadia's proprietary molecule, pimavanserin, a selective serotonin inverse agonist preferentially targeting 5-HT2a receptors. Acadia had partnered with Biovail in the late-stage clinical testing of the drug, which showed trial failures as of 2009. Acadia is running multiple Phase 2 and Phase 3 trials with pimavanserin in several central nervous system conditions: dementia-related psychosis, major depressive disorder, schizophrenia inadequate response, and schizophrenia ne ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
AbbVie
AbbVie is an American publicly traded biopharmaceutical company founded in 2013. It originated as a spin-off of Abbott Laboratories. History On October 19, 2011, Abbott Laboratories announced its plan to separate into two publicly traded companies. The new Abbott Laboratories would specialize in diversified products including medical devices, diagnostic equipment and nutrition products, while AbbVie would operate as a research-based pharmaceutical manufacturer. The separation was effective January 1, 2013, and AbbVie was officially listed on the New York Stock Exchange (ABBV) on January 2, 2013. According to Miles White, CEO at the time, the purpose of the split was to allow markets to value the two businesses separately. Some investors were concerned that the split was done to protect the value of the device business from the loss of value facing the drug division due to the imminent expiration of patents on Humira, which accounted for about half of the drug division's revenue. ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
National Organization For Rare Disorders
The National Organization for Rare Disorders (NORD) is an American non-profit organization aiming to provide support for individuals with rare diseases by advocating and funding research, education, and networking among service providers. It was founded in 1983 by Abbey Meyers, along with individuals and rare diseases leaders of rare disease support groups, and it is a 501(c)3 tax exempt organization. History The organization grew out of an "informal coalition" of support groups and families called together in the late 1970s to advocate legislation supporting development of orphan drugs, or drugs for treating rare diseases. They succeeded in getting the United States Congress to pass the Orphan Drug Act (ODA) in early 1983. The initial coalition was led by Abbey Meyers, whose son had Tourette syndrome. Tourette syndrome was estimated by the National Institutes of Health to affect 100,000 people in the United States. Meyers' son was helped by an experimental drug that the manufa ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Clinical Research
Clinical research is a branch of healthcare science that determines the safety and effectiveness ( efficacy) of medications, devices, diagnostic products and treatment regimens intended for human use. These may be used for prevention, treatment, diagnosis or for relieving symptoms of a disease. Clinical research is different from clinical practice. In clinical practice established treatments are used, while in clinical research evidence is collected to establish a treatment. Overview The term "clinical research" refers to the entire bibliography of a drug/device/biologic, in fact any test article from its inception in the lab to its introduction to the consumer market and beyond. Once the promising candidate or the molecule is identified in the lab, it is subjected to pre-clinical studies or animal studies where different aspects of the test article (including its safety toxicity if applicable and efficacy, if possible at this early stage) are studied. In the United States ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Biotechnology Companies
Biotechnology is the integration of natural sciences and engineering sciences in order to achieve the application of organisms, cells, parts thereof and molecular analogues for products and services. The term ''biotechnology'' was first used by Károly Ereky in 1919, meaning the production of products from raw materials with the aid of living organisms. Definition The concept of biotechnology encompasses a wide range of procedures for modifying living organisms according to human purposes, going back to domestication of animals, cultivation of the plants, and "improvements" to these through breeding programs that employ artificial selection and hybridization. Modern usage also includes genetic engineering as well as cell and tissue culture technologies. The American Chemical Society defines biotechnology as the application of biological organisms, systems, or processes by various industries to learning about the science of life and the improvement of the value of materials a ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Pharmaceutical Companies
The pharmaceutical industry discovers, develops, produces, and markets drugs or pharmaceutical drugs for use as medications to be administered to patients (or self-administered), with the aim to cure them, vaccinate them, or alleviate symptoms. Pharmaceutical companies may deal in generic or brand medications and medical devices. They are subject to a variety of laws and regulations that govern the patenting, testing, safety, efficacy using drug testing and marketing of drugs. The global pharmaceuticals market produced treatments worth $1,228.45 billion in 2020 and showed a compound annual growth rate (CAGR) of 1.8%. History Mid-1800s – 1945: From botanicals to the first synthetic drugs The modern era of pharmaceutical industry began with local apothecaries that expanded from their traditional role of distributing botanical drugs such as morphine and quinine to wholesale manufacture in the mid-1800s, and from discoveries resulting from applied research. Intentional drug d ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Europe
Europe is a large peninsula conventionally considered a continent in its own right because of its great physical size and the weight of its history and traditions. Europe is also considered a Continent#Subcontinents, subcontinent of Eurasia and it is located entirely in the Northern Hemisphere and mostly in the Eastern Hemisphere. Comprising the westernmost peninsulas of Eurasia, it shares the continental landmass of Afro-Eurasia with both Africa and Asia. 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 is commonly considered to be Boundaries between the continents of Earth#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 waterways of the Turkish Straits. "Europe" (pp. 68–69); "Asia" (pp. 90–91): "A commonly accepted division between Asia and E ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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