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Marathon Pharmaceuticals
Marathon Pharmaceuticals LLC was a privately held biopharmaceuticals company focused on drugs for people with rare diseases. The Illinois-based company developed and manufactured therapeutics and brought them to market. It employed 100 people in four global locations. In 2017, PTC Therapeutics acquired rights to Marathon Pharmaceuticals' drug Emflaza (deflazacort) for $140 million after criticism about their plan to sell the drug at a list price of $89,000 per year to sufferers despite the fact that the same drug was available in Canada and the UK for around $1,000 per year. Business model Marathon produced medicines for high-need, small patient populations, including patients with rare diseases as outlined by the U.S. Food and Drug Administration’s (FDA) Orphan Drug Act. The Act defines rare diseases as impacting fewer than 200,000 patients in the U.S. Marathon developed late-stage drugs, earned regulatory approvals, and then manufactured and commercialized medicines w ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
City
A city is a human settlement of notable size.Goodall, B. (1987) ''The Penguin Dictionary of Human Geography''. London: Penguin.Kuper, A. and Kuper, J., eds (1996) ''The Social Science Encyclopedia''. 2nd edition. London: Routledge. It can be defined as a permanent and densely settled place with administratively defined boundaries whose members work primarily on non-agricultural tasks. Cities generally have extensive systems for housing, transportation, sanitation, utilities, land use, production of goods, and communication. Their density facilitates interaction between people, government organisations and businesses, sometimes benefiting different parties in the process, such as improving efficiency of goods and service distribution. Historically, city-dwellers have been a small proportion of humanity overall, but following two centuries of unprecedented and rapid urbanization, more than half of the world population now lives in cities, which has had profound consequ ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Muscular Disorders, Atrophic
Muscle atrophy is the loss of skeletal muscle mass. It can be caused by immobility, aging, malnutrition, medications, or a wide range of injuries or diseases that impact the musculoskeletal or nervous system. Muscle atrophy leads to muscle weakness and causes disability. Disuse causes rapid muscle atrophy and often occurs during injury or illness that requires immobilization of a limb or bed rest. Depending on the duration of disuse and the health of the individual, this may be fully reversed with activity. Malnutrition first causes fat loss but may progress to muscle atrophy in prolonged starvation and can be reversed with nutritional therapy. In contrast, cachexia is a wasting syndrome caused by an underlying disease such as cancer that causes dramatic muscle atrophy and cannot be completely reversed with nutritional therapy. Sarcopenia is age-related muscle atrophy and can be slowed by exercise. Finally, diseases of the muscles such as muscular dystrophy or myopathies can cause ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Pharmaceutical Companies Of The United States
A medication (also called medicament, medicine, pharmaceutical drug, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease. Drug therapy (pharmacotherapy) is an important part of the medical field and relies on the science of pharmacology for continual advancement and on pharmacy for appropriate management. Drugs are classified in multiple ways. One of the key divisions is by level of control, which distinguishes prescription drugs (those that a pharmacist dispenses only on the order of a physician, physician assistant, or qualified nurse) from over-the-counter drugs (those that consumers can order for themselves). Another key distinction is between traditional small molecule drugs, usually derived from chemical synthesis, and biopharmaceuticals, which include recombinant proteins, vaccines, blood products used therapeutically (such as IVIG), gene therapy, monoclonal antibodies and cell therapy (for instance, stem cell therapies). Other ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Elijah Cummings
Elijah Eugene Cummings (January 18, 1951October 17, 2019) was an American politician and civil rights advocate who served in the United States House of Representatives for from 1996 until his death in 2019, when he was succeeded by his predecessor Kweisi Mfume. The district he represented included over half of the city of Baltimore, including most of the majority-black precincts of Baltimore County, and most of Howard County, Maryland. A member of the Democratic Party, Cummings previously served in the Maryland House of Delegates from 1983 to 1996. Cummings served as the chair of the Committee on Oversight and Reform from January 2019 until his death in October of the same year. Early life, education, and career Cummings was born on January 18, 1951, in Baltimore, son of Ruth Elma () and Robert Cummings. His parents were sharecroppers. He was the third child of seven. When he was 11 years old, Cummings and some friends worked to integrate a segregated swimming pool in S ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Bernie Sanders
Bernard Sanders (born September8, 1941) is an American politician who has served as the junior United States senator from Vermont since 2007. He was the U.S. representative for the state's at-large congressional district from 1991 to 2007. Sanders is the longest-serving independent in U.S. congressional history. He has a close relationship with the Democratic Party, having caucused with House and Senate Democrats for most of his congressional career. He is often seen as a leader of the democratic socialist movement in the United States. Sanders unsuccessfully sought the Democratic Party nomination for president of the United States in 2016 and 2020, finishing in second place in both campaigns. Before his election to Congress, he was mayor of Burlington, Vermont. Born into a working-class Jewish family and raised in the Brooklyn borough of New York City, Sanders attended Brooklyn College before graduating from the University of Chicago in 1964. While a student, ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Chicago Tribune
The ''Chicago Tribune'' is a daily newspaper based in Chicago, Illinois, United States, owned by Tribune Publishing. Founded in 1847, and formerly self-styled as the "World's Greatest Newspaper" (a slogan for which WGN radio and television are named), it remains the most-read daily newspaper in the Chicago metropolitan area and the Great Lakes region. It had the sixth-highest circulation for American newspapers in 2017. In the 1850s, under Joseph Medill, the ''Chicago Tribune'' became closely associated with the Illinois politician Abraham Lincoln, and the Republican Party's progressive wing. In the 20th century under Medill's grandson, Robert R. McCormick, it achieved a reputation as a crusading paper with a decidedly more American-conservative anti-New Deal outlook, and its writing reached other markets through family and corporate relationships at the ''New York Daily News'' and the '' Washington Times-Herald.'' The 1960s saw its corporate parent owner, Tribune Company ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
The Wall Street Journal
''The Wall Street Journal'' is an American business-focused, international daily newspaper based in New York City, with international editions also available in Chinese and Japanese. The ''Journal'', along with its Asian editions, is published six days a week by Dow Jones & Company, a division of News Corp. The newspaper is published in the broadsheet format and online. The ''Journal'' has been printed continuously since its inception on July 8, 1889, by Charles Dow, Edward Jones, and Charles Bergstresser. The ''Journal'' is regarded as a newspaper of record, particularly in terms of business and financial news. The newspaper has won 38 Pulitzer Prizes, the most recent in 2019. ''The Wall Street Journal'' is one of the largest newspapers in the United States by circulation, with a circulation of about 2.834million copies (including nearly 1,829,000 digital sales) compared with '' USA Today''s 1.7million. The ''Journal'' publishes the luxury news and lifestyle magazine ' ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Rare Disease
A rare disease is any disease that affects a small percentage of the population. In some parts of the world, an orphan disease is a rare disease whose rarity means there is a lack of a market large enough to gain support and resources for discovering treatments for it, except by the government granting economically advantageous conditions to creating and selling such treatments. Orphan drugs are ones so created or sold. Most rare diseases are genetic and thus are present throughout the person's entire life, even if symptoms do not immediately appear. Many rare diseases appear early in life, and about 30% of children with rare diseases will die before reaching their fifth birthdays. With only four diagnosed patients in 27 years, ribose-5-phosphate isomerase deficiency is considered the rarest known genetic disease. No single cut-off number has been agreed upon for which a disease is considered rare. A disease may be considered rare in one part of the world, or in a particular gr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Deflazacort
Deflazacort (trade name Calcort among others) is a glucocorticoid used as an anti-inflammatory and immunomodulatory agent. It was patented in 1965 and approved for medical use in 1985. The U.S. Food and Drug Administration (FDA) considers it to be a first-in-class medication for Duchenne Muscular Dystrophy. Medical uses The manufacturer lists the following uses for deflazacort: In the United States, deflazacort is approved for the treatment of duchenne muscular dystrophy in people over the age of two. Adverse effects Deflazacort carries the risks common to all corticosteroids, including immune suppression, decreased bone density, and endocrine insufficiency. In clinical trials, the most common side effects (>10% above placebo) were Cushing's-like appearance, weight gain, and increased appetite. Pharmacology Mechanism of action Deflazacort is an inactive prodrug which is metabolized rapidly to the active drug 21-desacetyldeflazacort. Relative potency Deflazacort's ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy that primarily affects boys. Muscle weakness usually begins around the age of four, and worsens quickly. Muscle loss typically occurs first in the thighs and pelvis followed by the arms. This can result in trouble standing up. Most are unable to walk by the age of 12. Affected muscles may look larger due to increased fat content. Scoliosis is also common. Some may have intellectual disability. Females with a single copy of the defective gene may show mild symptoms. The disorder is X-linked recessive. About two thirds of cases are inherited from a person's mother, while one third of cases are due to a new mutation. It is caused by a mutation in the gene for the protein dystrophin. Dystrophin is important to maintain the muscle fiber's cell membrane. Genetic testing can often make the diagnosis at birth. Those affected also have a high level of creatine kinase in their blood. Although there is no know ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Blood Disorders
Hematologic diseases are disorders which primarily affect the blood & blood-forming organs. Hematologic diseases include rare genetic disorders, anemia, HIV, sickle cell disease & complications from chemotherapy or transfusions. Myeloid * Hemoglobinopathies (congenital abnormality of the hemoglobin molecule or of the rate of hemoglobin synthesis) ** Sickle cell disease ** Thalassemia ** Methemoglobinemia * Anemias (lack of red blood cells or hemoglobin) ** Iron-deficiency anemia ** Megaloblastic anemia *** Vitamin B12 deficiency **** Pernicious anemia *** Folate deficiency ** Hemolytic anemias (destruction of red blood cells) *** Genetic disorders of RBC membrane **** Hereditary spherocytosis **** Hereditary elliptocytosis **** Congenital dyserythropoietic anemia *** Genetic disorders of RBC metabolism **** Glucose-6-phosphate dehydrogenase deficiency (G6PD) **** Pyruvate kinase deficiency *** Immune mediated hemolytic anemia ( direct Coombs test is positive) **** Autoimmune he ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Gastrointestinal Disorder
Gastrointestinal diseases (abbrev. GI diseases or GI illnesses) refer to diseases involving the gastrointestinal tract, namely the oesophagus, stomach, small intestine, large intestine and rectum, and the accessory organs of digestion, the liver, gallbladder, and pancreas. Oral disease The oral cavity is part of the gastrointestinal system and as such the presence of alterations in this district can be the first sign of both systemic and gastrointestinal diseases. By far the most common oral conditions are plaque-induced diseases (e.g., gingivitis, periodontitis, dental caries). Oral symptoms can be similar to lesions occurring elsewhere in the digestive tract, with a pattern of swelling, inflammation, ulcers, and fissures. If these signs are present, then patients are more likely to also have anal and esophageal lesions and experience other extra-intestinal disease manifestations. Some diseases which involve other parts of the GI tract can manifest in the mouth, alone or i ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
