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Alipogene Tiparvovec
Alipogene tiparvovec, sold under the brand name Glybera, is a gene therapy treatment designed to reverse lipoprotein lipase deficiency (LPLD), a rare recessive disorder, due to mutations in LPL, which can cause severe pancreatitis. It was recommended for approval by the European Medicines Agency in July 2012 and approved by the European Commission in November of the same year. It was the first marketing authorisation for a gene therapy treatment in either Europe or the United States. The drug is administered via a series of injections into the leg muscles – as many as 60, all in one session. It is a one-time treatment intended to last at least ten years. Glybera gained infamy as the "million-dollar drug" and proved commercially unsuccessful for a number of reasons. Its cost to patients and payers, together with the rarity of LPLD, high maintenance costs to its manufacturer , and failure to achieve approval in the US, led to withdrawing the drug after two years on the EU m ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Intramuscular Injection
Intramuscular injection, often abbreviated IM, is the injection of a substance into a muscle. In medicine, it is one of several methods for parenteral administration of medications. Intramuscular injection may be preferred because muscles have larger and more numerous blood vessels than subcutaneous tissue, leading to faster absorption than subcutaneous or intradermal injections. Medication administered via intramuscular injection is not subject to the first-pass metabolism effect which affects oral medications. Common sites for intramuscular injections include the deltoid muscle of the upper arm and the gluteal muscle of the buttock. In infants, the vastus lateralis muscle of the thigh is commonly used. The injection site must be cleaned before administering the injection, and the injection is then administered in a fast, darting motion to decrease the discomfort to the individual. The volume to be injected in the muscle is usually limited to 2–5 milliliters, depending on in ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Saguenay River
__NOTOC__ The Saguenay River () is a major river of Quebec, Canada. It drains Lac Saint-Jean in the Laurentian Highlands, leaving at Alma and running east; the city of Saguenay is located on the river. It drains into the Saint Lawrence River. Tadoussac, founded as a French colonial trading post in 1600, is located on the northeast bank at this site. The river has a very high flow-rate and is bordered by steep cliffs associated with the Saguenay Graben. Tide waters flow in its fjord upriver as far as Chicoutimi (about 100 kilometres). Many Beluga whales breed in the cold waters at its mouth, making Tadoussac a popular site for whale watching and sea kayaking; Greenland sharks also frequent the depths of the river. The area of the confluence of the Saguenay and Saint Lawrence is protected by the Saguenay–St. Lawrence Marine Park, one of Canada's national parks. History The Saguenay River was used as an important trade route into the interior for the First Nations people of ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Health Care Costs
Health, according to the World Health Organization, is "a state of complete physical, mental and social well-being and not merely the absence of disease and infirmity".World Health Organization. (2006)''Constitution of the World Health Organization''– ''Basic Documents'', Forty-fifth edition, Supplement, October 2006. A variety of definitions have been used for different purposes over time. Health can be promoted by encouraging healthful activities, such as regular physical exercise and adequate sleep, and by reducing or avoiding unhealthful activities or situations, such as smoking or excessive stress. Some factors affecting health are due to individual choices, such as whether to engage in a high-risk behavior, while others are due to structural causes, such as whether the society is arranged in a way that makes it easier or harder for people to get necessary healthcare services. Still, other factors are beyond both individual and group choices, such as genetic disorders. ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
List Of Gene Therapies
This article contains a list of commercially available gene therapies. Gene therapies * Alipogene tiparvovec (Glybera): AAV-based treatment for lipoprotein lipase deficiency (no longer commercially available) * Axicabtagene ciloleucel (Yescarta): treatment for large B-cell lymphoma * Betibeglogene autotemcel (Zynteglo): treatment for beta thalassemia * Brexucabtagene autoleucel (Tecartus): treatment for mantle cell lymphoma and acute lymphoblastic leukemia * Cambiogenplasmid (Neovasculgen): treatment for vascular endothelial growth factor peripheral artery disease * Ciltacabtagene autoleucel (Carvykti): treatment for multiple myeloma * Elivaldogene autotemcel (Skysona): treatment for cerebral adrenoleukodystrophy * Etranacogene dezaparvovec (Hemgenix): treatment for hemophilia B * Gendicine: treatment for head and neck squamous cell carcinoma * Idecabtagene vicleucel (Abecma): treatment for multiple myeloma * Nadofaragene firadenovec (Adstiladrin): treatment for bladder c ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Antibody-dependent Cellular Cytotoxicity
Antibody-dependent cellular cytotoxicity (ADCC), also referred to as antibody-dependent cell-mediated cytotoxicity, is a mechanism of cell-mediated immune defense whereby an effector cell of the immune system actively lyses a target cell, whose membrane-surface antigens have been bound by specific antibodies. It is one of the mechanisms through which antibodies, as part of the humoral immune response, can act to limit and contain infection. ADCC is independent of the immune complement system that also lyses targets but does not require any other cell. ADCC requires an effector cell which classically is known to be natural killer (NK) cells that typically interact with immunoglobulin G (IgG) antibodies. However, macrophages, neutrophils and eosinophils can also mediate ADCC, such as eosinophils killing certain parasitic worms known as helminths via IgE antibodies. In general, ADCC has typically been described as the immune response to antibody-coated cells leading ultimatel ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Antibodies
An antibody (Ab), also known as an immunoglobulin (Ig), is a large, Y-shaped protein used by the immune system to identify and neutralize foreign objects such as pathogenic bacteria and viruses. The antibody recognizes a unique molecule of the pathogen, called an antigen. Each tip of the "Y" of an antibody contains a paratope (analogous to a lock) that is specific for one particular epitope (analogous to a key) on an antigen, allowing these two structures to bind together with precision. Using this binding mechanism, an antibody can ''tag'' a microbe or an infected cell for attack by other parts of the immune system, or can neutralize it directly (for example, by blocking a part of a virus that is essential for its invasion). To allow the immune system to recognize millions of different antigens, the antigen-binding sites at both tips of the antibody come in an equally wide variety. In contrast, the remainder of the antibody is relatively constant. It only occurs in a few vari ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Immunogenicity
Immunogenicity is the ability of a foreign substance, such as an antigen, to provoke an immune response in the body of a human or other animal. It may be wanted or unwanted: * Wanted immunogenicity typically relates to vaccines, where the injection of an antigen (the vaccine) provokes an immune response against the pathogen, protecting the organism from future exposure. Immunogenicity is a central aspect of vaccine development. * Unwanted immunogenicity is an immune response by an organism against a therapeutic antigen. This reaction leads to production of anti-drug-antibodies (ADAs), inactivating the therapeutic effects of the treatment and potentially inducing adverse effects. A challenge in biotherapy is predicting the immunogenic potential of novel protein therapeutics. For example, immunogenicity data from high-income countries are not always transferable to low-income and middle-income countries. Another challenge is considering how the immunogenicity of vaccines changes with ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Cancer
Cancer is a group of diseases involving abnormal cell growth with the potential to invade or spread to other parts of the body. These contrast with benign tumors, which do not spread. Possible signs and symptoms include a lump, abnormal bleeding, prolonged cough, unexplained weight loss, and a change in bowel movements. While these symptoms may indicate cancer, they can also have other causes. Over 100 types of cancers affect humans. Tobacco use is the cause of about 22% of cancer deaths. Another 10% are due to obesity, poor diet, lack of physical activity or excessive drinking of alcohol. Other factors include certain infections, exposure to ionizing radiation, and environmental pollutants. In the developing world, 15% of cancers are due to infections such as ''Helicobacter pylori'', hepatitis B, hepatitis C, human papillomavirus infection, Epstein–Barr virus and human immunodeficiency virus (HIV). These factors act, at least partly, by changing the genes of ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Retrovirus
A retrovirus is a type of virus that inserts a DNA copy of its RNA genome into the DNA of a host cell that it invades, thus changing the genome of that cell. Once inside the host cell's cytoplasm, the virus uses its own reverse transcriptase enzyme to produce DNA from its RNA genome, the reverse of the usual pattern, thus ''retro'' (backwards). The new DNA is then incorporated into the host cell genome by an integrase enzyme, at which point the retroviral DNA is referred to as a provirus. The host cell then treats the viral DNA as part of its own genome, transcribing and translating the viral genes along with the cell's own genes, producing the proteins required to assemble new copies of the virus. Although retroviruses have different subfamilies, they have three basic groups: the oncoretroviruses (oncogenic retroviruses), the lentiviruses (slow retroviruses) and the spumaviruses (foamy viruses). The oncoretroviruses are able to cause cancer in some species, the lentiviru ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Immunosuppressive Therapy
Immunosuppression is a reduction of the activation or efficacy of the immune system. Some portions of the immune system itself have immunosuppressive effects on other parts of the immune system, and immunosuppression may occur as an adverse reaction to treatment of other conditions. In general, deliberately induced immunosuppression is performed to prevent the body from rejecting an organ transplant. Additionally, it is used for treating graft-versus-host disease after a bone marrow transplant, or for the treatment of auto-immune diseases such as systemic lupus erythematosus, rheumatoid arthritis, Sjögren's syndrome, or Crohn's disease. This is typically done using medications, but may involve surgery (splenectomy), plasmapheresis, or radiation. A person who is undergoing immunosuppression, or whose immune system is weak for some other reasons (such as chemotherapy or HIV), is said to be ''immunocompromised''. Deliberately induced Administration of immunosuppressive medi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Adeno-associated Virus
Adeno-associated viruses (AAV) are small viruses that infect humans and some other primate species. They belong to the genus ''Dependoparvovirus'', which in turn belongs to the family ''Parvoviridae''. They are small (approximately 26 nm in diameter) replication-defective, nonenveloped viruses and have linear single-stranded DNA (ssDNA) genome of approximately 4.8 kilobases (kb). AAV are not currently known to cause disease. The viruses cause a very mild immune response. Several additional features make AAV an attractive candidate for creating viral vectors for gene therapy, and for the creation of isogenic human disease models. Gene therapy vectors using AAV can infect both dividing and quiescent cells and persist in an extrachromosomal state without integrating into the genome of the host cell. In the native virus, however, integration of virally carried genes into the host genome does occur. Integration can be important for certain applications, but can also have unwan ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Chiesi Farmaceutici
Chiesi Farmaceutici S.p.A. is an Italian family controlled global pharmaceutical company based in Parma, Emilia-Romagna. Chiesi has 30 affiliates in the world, nearly 6,389 total employees and provides medicines to patients in 90 nations. Chiesi currently has revenues of 2.229 billion euros. According to 2020 official data from the European Patent Office (EPO), Chiesi Group, with 38 patents filed, is confirmed the first Italian Pharmaceutical Company and fourth among Italian Companies across all sectors for filing the highest number of patents. In June 2019, Chiesi became a certified B Corporation, thus choosing to adopt a new legal status under US and Italian law. This ensures Chiesi a legal protection to pursue a business model placing social and environmental values on equal footing with profits. Following the acquisition, in 1999, of Huddersfield based Trinity Pharmaceuticals, Chiesi has also had a significant presence in Manchester, United Kingdom. History Established i ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
