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National Hemophilia Foundation
The National Bleeding Disorders Foundation (NBDF) is a United States patient advocacy organization for the care and treatment of inheritable blood and bleeding disorders such as hemophilia and von Willebrand disease. Founded in 1948, NBDF, then known as the National Hemophilia Foundation, helps secure funding for treatment centers, and develops national guidelines for treatment and health care policy. The organization also serves as a central point for information on the disorders. History The National Bleeding Disorders Foundation was founded in 1948, as the first national hemophilia advocacy organization in the United States. One of its early initiatives was to secure funding for Comprehensive Hemophilia Diagnostic and Treatment Centers (HTC). In 1993, the foundation received media coverage of its efforts to hold health care company Baxter International accountable for infecting 10,000 hemophiliac members with AIDS due to HIV contaminated clotting products. In 1994, the NHF ...
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Haemophilia
Haemophilia, or hemophilia (), is a mostly inherited genetic disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding. This results in people bleeding for a longer time after an injury, easy bruising, and an increased risk of bleeding inside joints or the brain. Those with a mild case of the disease may have symptoms only after an accident or during surgery. Bleeding into a joint can result in permanent damage while bleeding in the brain can result in long term headaches, seizures, or a decreased level of consciousness. There are two main types of haemophilia: haemophilia A, which occurs due to low amounts of clotting factor VIII, and haemophilia B, which occurs due to low levels of clotting factor IX. They are typically inherited from one's parents through an X chromosome carrying a nonfunctional gene. Rarely a new mutation may occur during early development or haemophilia may develop later in life due to antibodies forming against ...
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Public Policy (law)
In private international law, the public policy doctrine or (French: "public order") concerns the body of principles that underpin the operation of legal systems in each state. This addresses the social, moral and economic values that tie a society together: values that vary in different cultures and change over time. Law regulates behaviour either to reinforce existing social expectations or to encourage constructive change, and laws are most likely to be effective when they are consistent with the most generally accepted societal norms and reflect the collective morality of the society. In performing this function, Cappalli has suggested that the critical values of any legal system include impartiality, neutrality, certainty, equality, openness, flexibility, and growth. This assumes that a state's courts function as '' dispute resolution systems'', which avoid the violence that often otherwise accompanies private resolution of disputes. That is, citizens have to be encourag ...
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Education
Education is a purposeful activity directed at achieving certain aims, such as transmitting knowledge or fostering skills and character traits. These aims may include the development of understanding, rationality, kindness, and honesty. Various researchers emphasize the role of critical thinking in order to distinguish education from indoctrination. Some theorists require that education results in an improvement of the student while others prefer a value-neutral definition of the term. In a slightly different sense, education may also refer, not to the process, but to the product of this process: the mental states and dispositions possessed by educated people. Education originated as the transmission of cultural heritage from one generation to the next. Today, educational goals increasingly encompass new ideas such as the liberation of learners, skills needed for modern society, empathy, and complex vocational skills. Types of education are commonly divided into formal ...
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Hemophilia
Haemophilia, or hemophilia (), is a mostly inherited genetic disorder that impairs the body's ability to make blood clots, a process needed to stop bleeding. This results in people bleeding for a longer time after an injury, easy bruising, and an increased risk of bleeding inside joints or the brain. Those with a mild case of the disease may have symptoms only after an accident or during surgery. Bleeding into a joint can result in permanent damage while bleeding in the brain can result in long term headaches, seizures, or a decreased level of consciousness. There are two main types of haemophilia: haemophilia A, which occurs due to low amounts of clotting factor VIII, and haemophilia B, which occurs due to low levels of clotting factor IX. They are typically inherited from one's parents through an X chromosome carrying a nonfunctional gene. Rarely a new mutation may occur during early development or haemophilia may develop later in life due to antibodies forming against ...
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Von Willebrand Disease
Von Willebrand disease (VWD) is the most common hereditary blood-clotting disorder in humans. An acquired form can sometimes result from other medical conditions. It arises from a deficiency in the quality or quantity of von Willebrand factor (VWF), a multimeric protein that is required for platelet adhesion. It is known to affect several breeds of dogs as well as humans. The three forms of VWD are hereditary, acquired, and pseudo or platelet type. The three types of hereditary VWD are VWD type 1, VWD type 2, and VWD type 3. Type 2 contains various subtypes. Platelet type VWD is also an inherited condition. In 2008 a new diagnostic category of "Low VWF" was proposed to include those individuals whose von Willebrand factor levels were below the normal reference range but not low enough to be von Willebrand disease (levels in the 30-50 IU/dL range). Patients with low VWF can experience bleeding, despite mild reductions in VWF levels. VWD type 1 is the most common type of the dis ...
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Baxter International
Baxter International Inc. is an American multinational healthcare company with headquarters in Deerfield, Illinois. The company primarily focuses on products to treat kidney disease, and other chronic and acute medical conditions. The company had 2017 sales of $10.6 billion, across two businesses: BioScience and Medical Products. Baxter's BioScience business produces recombinant and blood plasma proteins to treat hemophilia and other bleeding disorders; plasma-based therapies to treat immune deficiencies and other chronic and acute blood-related conditions; products for regenerative medicine, and vaccines. Baxter's Medical Products business produces intravenous products and other products used in the delivery of fluids and drugs to patients; inhalational anaesthetics; contract manufacturing services; and products to treat end-stage kidney disease, or irreversible kidney failure, including products for peritoneal dialysis and hemodialysis. History Baxter International was fou ...
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Parvovirus B19
Primate erythroparvovirus 1, generally referred to as B19 virus (B19V), parvovirus B19 or sometimes erythrovirus B19, is the first (and until 2005 the only) known human virus in the family ''Parvoviridae'', genus ''Erythroparvovirus''; it measures only 23–26  nm in diameter. The name is derived from Latin, parvum meaning small, reflecting the fact that B19 ranks among the smallest DNA viruses. B19 virus is most known for causing disease in the pediatric population; however, it can also affect adults. It is the classic cause of the childhood rash called fifth disease or erythema infectiosum, or "slapped cheek syndrome". The virus was discovered by chance in 1975 by Australian virologist Yvonne Cossart. It gained the B19 name because it was discovered in well B19 of a large series of microtiter plates. Virology Erythroviruses belong to the ''Parvoviridae'' family of small DNA viruses. Human parvovirus B19 is a non-enveloped, icosahedral virus that contains a single-stranded ...
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Hepatitis A
Hepatitis A is an infectious disease of the liver caused by ''Hepatovirus A'' (HAV); it is a type of viral hepatitis. Many cases have few or no symptoms, especially in the young. The time between infection and symptoms, in those who develop them, is 2–6 weeks. When symptoms occur, they typically last 8 weeks and may include nausea, vomiting, diarrhea, jaundice, fever, and abdominal pain. Around 10–15% of people experience a recurrence of symptoms during the 6 months after the initial infection. Acute liver failure may rarely occur, with this being more common in the elderly. It is usually spread by eating food or drinking water contaminated with infected feces. Undercooked or raw shellfish are relatively common sources. It may also be spread through close contact with an infectious person. While children often do not have symptoms when infected, they are still able to infect others. After a single infection, a person is immune for the rest of his or her life. Diagnosis requir ...
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Alex Borstein
Alexandrea Borstein (born February 15, 1971) is an American actress, comedian, writer and producer. Borstein voices Lois Griffin on the animated comedy television series '' Family Guy'' (1999–present), and won a Primetime Emmy Award for the role. She gained acclaim for starring as Susie Myerson in the comedy-drama series ''The Marvelous Mrs. Maisel'' (2017–present), which has earned her two Primetime Emmy Awards. Borstein also had lead roles as various characters on the sketch comedy series ''MADtv'' (1997–2009), as well as Dawn Forchette in the medical comedy series '' Getting On'' (2013–2015). She had supporting roles in numerous films, including ''The Lizzie McGuire Movie'', ''Catwoman'' (2004), ''Good Night, and Good Luck'' (2005), ''Dinner for Schmucks'' (2010), ''Ted'' (2012), ''ParaNorman'' (2012), and '' A Million Ways to Die in the West'' (2014). Borstein spent her childhood in Deerfield, Illinois, a suburb of Chicago, before moving with her family to Northridge ...
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Genetic Testing
Genetic testing, also known as DNA testing, is used to identify changes in DNA sequence or chromosome structure. Genetic testing can also include measuring the results of genetic changes, such as RNA analysis as an output of gene expression, or through biochemical analysis to measure specific protein output. In a medical setting, genetic testing can be used to diagnose or rule out suspected genetic disorders, predict risks for specific conditions, or gain information that can be used to customize medical treatments based on an individual's genetic makeup. Genetic testing can also be used to determine biological relatives, such as a child's biological parentage (genetic mother and father) through DNA paternity testing, or be used to broadly predict an individual's ancestry. Genetic testing of plants and animals can be used for similar reasons as in humans (e.g. to assess relatedness/ancestry or predict/diagnose genetic disorders), to gain information used for selective breeding, ...
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Risk Evaluation And Mitigation Strategies
Risk Evaluation and Mitigation Strategies (REMS) is a program of the US Food and Drug Administration for the monitoring of medications with a high potential for serious adverse effects. REMS applies only to specific prescription drugs, but can apply to brand name or generic drugs. The REMS program was formalized in 2007. The FDA determines as part of the drug approvals process that a REMS is necessary, and the drug company develops and maintains the individual program. REMS applies only to specific prescription drugs, but can apply to brand name or generic drugs. REMS for generic drugs may be created in collaboration with the manufacture of the brand name drug. The FDA may remove the REMS requirement if it is found to not improve patient safety. The REMS program developed out of previous systems dating back to the 1980s for monitoring the use of a small number of high-risk drugs such as the Accutane, which causes serious birth defects, Clozaril, which can cause agranulocytosis, a ...
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Valoctocogene Roxaparvovec
Valoctocogene roxaparvovec, sold under the brand name Roctavian, is a gene therapy used for the treatment of hemophilia A. It was developed by BioMarin Pharmaceutical. Valoctocogene roxaparvovec is made of a virus (AAV5) that has been modified to contain the gene for factor VIII, which is lacking in people with hemophilia A. It is given by intravenous infusion. The most common side effects include increased levels of the liver enzymes alanine aminotransferase and aspartate aminotransferase (signs of possible liver problems), increased levels of the enzyme lactate dehydrogenase (sign of possible tissue damage), nausea (feeling sick), and headache. Valoctocogene roxaparvovec was approved for medical use in the European Union in August 2022, and in the United States in June 2023. Medical uses Valoctocogene roxaparvovec is indicated for the treatment of severe haemophilia A (congenital factor VIII deficiency) in adults without a history of factor VIII inhibitors and without dete ...
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