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Rare Diseases Act Of 2002
The Rare Disease Act of 2002 is a law passed in the United States that establishes the statutory authorization for the Office of Rare Diseases as a federal entity able to recommend a national research agenda, coordinate research, and provide educational activities for researchers. Background A rare disease or disorder is defined in the U.S. as one affecting fewer than 200,000 Americans. There are more than 6,000 known rare diseases, and it is estimated that about 25 million Americans are affected by them (as of 2002). Prior to the RDA was the Orphan Drug Act of 1983 which was designed to facilitate the development and commercialization of drugs to treat rare diseases, termed ''orphan drugs''. This act however did not provide for the creation of a centralized structure able to coordinate research or recommend agendas that would better facilitate research and education. Legislative history S. 1379 (Rare Diseases Act of 2001) was introduced in 2001 by Sen. Edward Kennedy (D-MA) bu ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Public Health And Social Welfare
In public relations and communication science, publics are groups of individual people, and the public (a.k.a. the general public) is the totality of such groupings. This is a different concept to the sociological concept of the ''Öffentlichkeit'' or public sphere. The concept of a public has also been defined in political science, psychology, marketing, and advertising. In public relations and communication science, it is one of the more ambiguous concepts in the field. Although it has definitions in the theory of the field that have been formulated from the early 20th century onwards, and suffered more recent years from being blurred, as a result of conflation of the idea of a public with the notions of audience, market segment, community, constituency, and stakeholder. Etymology and definitions The name "public" originates with the Latin '' publicus'' (also '' poplicus''), from '' populus'', to the English word ' populace', and in general denotes some mass population ("the ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Ted Kennedy
Edward Moore Kennedy (February 22, 1932 – August 25, 2009) was an American lawyer and politician who served as a United States senator from Massachusetts for almost 47 years, from 1962 until his death in 2009. A member of the Democratic Party and the prominent political Kennedy family, he was the second most senior member of the Senate when he died. He is ranked fifth in United States history for length of continuous service as a senator. Kennedy was the younger brother of President John F. Kennedy and U.S. attorney general and U.S. senator Robert F. Kennedy. He was the father of Congressman Patrick J. Kennedy. After attending Harvard University and earning his law degree from the University of Virginia, Kennedy began his career as an assistant district attorney in Suffolk County, Massachusetts. Kennedy was 30 years old when he first entered the Senate, winning a November 1962 special election in Massachusetts to fill the vacant seat previously held by his broth ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Rare Diseases
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 gro ... [...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 manuf ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Rare Disease Day
Rare Disease Day is an observance held on the last day of February to raise awareness for rare diseases and improve access to treatment and medical representation for individuals with rare diseases and their families. The European Organisation for Rare Diseases established the day in 2008 to raise awareness for unknown or overlooked illnesses. According to that organization, treatment for many rare diseases is insufficient, as are the social networks to support individuals with rare diseases and their families; furthermore, while there were already numerous days dedicated to individuals with specific diseases (such as AIDS, cancer, etc.), there had previously not been a day for representing those affected by rare diseases. In 2009, Rare Disease Day went global as the National Organization for Rare Disorders mobilized 200 rare disease patient advocacy organizations in the United States while organizations in China, Australia, Taiwan, and Latin America also lead efforts in their r ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drugs
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy in many countries and has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development. In the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs which would otherwise lack sufficient profit motive to attract corporate research budgets and personnel. Definition According to the US Food and Drug Administration (FDA), an orphan drug is defined as one "intended for ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Data Sharing
Data sharing is the practice of making data used for scholarly research available to other investigators. Many funding agencies, institutions, and publication venues have policies regarding data sharing because transparency and openness are considered by many to be part of the scientific method. A number of funding agencies and science journals require authors of peer-reviewed papers to share any supplemental information ( raw data, statistical methods or source code) necessary to understand, develop or reproduce published research. A great deal of scientific research is not subject to data sharing requirements, and many of these policies have liberal exceptions. In the absence of any binding requirement, data sharing is at the discretion of the scientists themselves. In addition, in certain situations governments and institutions prohibit or severely limit data sharing to protect proprietary interests, national security, and subject/patient/victim confidentiality. Data sharing m ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Rare Diseases Clinical Research Network
The Rare Diseases Clinical Research Network (RDCRN) is an initiative of the Office of Rare Diseases Research (ORDR). RDCRN is funded by the ORDR, the National Center for Advancing Translational Sciences and collaborating institute centers. The RDCRN is designed to advance medical research on rare diseases by providing support for clinical studies and facilitating collaboration, study enrollment and data sharing. Through the RDCRN consortia, physician scientists and their multidisciplinary teams work together with patient advocacy groups to study more than 200 rare diseases at sites across the nation. Established by Congress under the Rare Diseases Act in 2002, the RDCRN has included more than 350 sites in the United States and more than 50 in 22 other countries. To date, they have encompassed 237 research protocols and included more than 56,000 participants in studies ranging from immune system disorders and rare cancers to heart and lung disorders, brain development diseases and mo ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
National Institutes Of Health
The National Institutes of Health, commonly referred to as NIH (with each letter pronounced individually), is the primary agency of the United States government responsible for biomedical and public health research. It was founded in the late 1880s and is now part of the United States Department of Health and Human Services. The majority of NIH facilities are located in Bethesda, Maryland, and other nearby suburbs of the Washington metropolitan area, with other primary facilities in the Research Triangle Park in North Carolina and smaller satellite facilities located around the United States. The NIH conducts its own scientific research through the NIH Intramural Research Program (IRP) and provides major biomedical research funding to non-NIH research facilities through its Extramural Research Program. , the IRP had 1,200 principal investigators and more than 4,000 postdoctoral fellows in basic, translational, and clinical research, being the largest biomedical research ins ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drugs
An orphan drug is a pharmaceutical agent developed to treat medical conditions which, because they are so rare, would not be profitable to produce without government assistance. The conditions are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy in many countries and has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development. In the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs which would otherwise lack sufficient profit motive to attract corporate research budgets and personnel. Definition According to the US Food and Drug Administration (FDA), an orphan drug is defined as one "intended for ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
John Shimkus
John Mondy Shimkus (, born February 21, 1958) is an American politician who served as a U.S. representative from 1997 to 2021, representing the 20th, 19th and 15th congressional districts of Illinois. Shimkus is a member of the Republican Party. On August 30, 2019, he announced that he would not seek re-election for his seat in 2020 and was succeeded by fellow Republican Mary Miller. Early life, education, and career Shimkus is a lifelong resident of Collinsville, part of the Metro East portion of the St. Louis metropolitan area. He is the son of Kathleen N. (née Mondy) and Gene L. Shimkus. His paternal grandfather was of Lithuanian descent. Shimkus earned his bachelor's degree at the United States Military Academy. After serving his five-year United States Army commitment, he entered the United States Army Reserve, retiring in 2008 as a lieutenant colonel. While in the U.S. Army, Shimkus earned the Expert Infantry Badge, Ranger Tab, and Parachutist Badge. He served oversea ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drug Act Of 1983
The Orphan Drug Act of 1983 is a law passed in the United States to facilitate development of orphan drugs—drugs for rare diseases such as Huntington's disease, myoclonus, ALS, Tourette syndrome and muscular dystrophy which affect small numbers of individuals residing in the United States. Orphan drug designation does not indicate that the therapeutic is either safe and effective or legal to manufacture and market in the United States. That process is handled through other offices in the US Food and Drug Administration. Instead, the designation means only that the sponsor qualifies for certain benefits from the federal government, such as market exclusivity and reduced taxes. In 1982 an informal coalition of supporters and families of patients with rare diseases who formed National Organization for Rare Disorders (NORD) and others, called for change to legislation to support development of orphan drugs, or drugs for treating rare diseases. They succeeded in getting the Unit ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
