Leniolisib
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Leniolisib
Leniolisib (INN), sold under the brand name Joenja, is a medication used for the treatment of activated phosphoinositide 3-kinase delta syndrome (APDS). It is a kinase inhibitor that is taken by mouth. The most common side effects include headache, sinusitis, and atopic dermatitis. Leniolisib was approved for medical use in the United States in March 2023. It is the first approved medication for the treatment of activated PI3K delta syndrome. The US Food and Drug Administration (FDA) considers it to be a first-in-class medication. Medical uses Leniolisib is indicated for the treatment of activated phosphoinositide 3-kinase delta syndrome (activated PI3K delta syndrome) in people twelve years of age and older. Activated PI3K delta syndrome is caused by mutations in either of two genes, PIK3CD or PIK3R1, that regulate the maturation of white blood cells, especially B cells and T cells. This leads to a decrease in immune cells, which makes it difficult for people with a ...
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Activated PI3K Delta Syndrome
Activated PI3K delta syndrome (APDS) is a primary immunodeficiency disease caused by activating mutation, gain of function mutations in the PIK3CD gene. Symptoms and signs The signs and symptoms of ''activated PI3K Delta Syndrome'' are consistent with the following: * Immunodeficiency * Lymphadenopathy * Sinopulmonary infections * Bronchiectasis Cause In terms of genetics, activated PI3K Delta Syndrome is autosomal dominant, a mutation in phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit delta isoform is the reason for this condition (located at chromosome 1p36.) Mechanism The pathophysiology of activated PI3K delta syndrome has several aspects. The normal function has P110δ (PI3K) involved in immune system regulation. P110δ effect is not limited to the immune system; P110δ has a ''presence'' in transformed epithelial cells and cell adhesion molecules (airway inflammation), and research has been done on the possibility of P110δ in the nervous system. Activa ...
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Phosphoinositide 3-kinase Inhibitor
Phosphoinositide 3-kinase inhibitors (PI3K inhibitors) are a class of medical drugs that are mainly used to treat advanced cancers. They function by inhibiting one or more of the phosphoinositide 3-kinase (PI3K) enzymes, which are part of the PI3K/AKT/mTOR pathway. This signal pathway regulates cellular functions such as growth and survival. It is strictly regulated in healthy cells, but is always active in many cancer cells, allowing the cancer cells to better survive and multiply. PI3K inhibitors block the PI3K/AKT/mTOR pathway and thus slow down cancer growth. They are examples of a targeted therapy. While PI3K inhibitors are an effective treatment, they can have very severe side effects and are therefore only used if other treatments have failed or are not suitable. After PI3K inhibitors had been under investigation as anti-cancer drugs for several years, the first one to be approved for treatment in clinical practice was idelalisib in 2014. Several others followed, and even ...
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By Mouth
Oral administration is a route of administration where a substance is taken through the mouth. Per os abbreviated to P.O. is sometimes used as a direction for medication to be taken orally. Many medications are taken orally because they are intended to have a systemic effect, reaching different parts of the body via the bloodstream, for example. Oral administration can be easier and less painful than other routes, such as injection. However, the onset of action is relatively low, and the effectiveness is reduced if it is not absorbed properly in the digestive system, or if it is broken down by digestive enzymes before it can reach the bloodstream. Some medications may cause gastrointestinal side effects, such as nausea or vomiting, when taken orally. Oral administration can also only be applied to conscious patients, and patients willing and able to swallow. Terminology ''Per os'' (; ''P.O.'') is an adverbial phrase meaning literally from Latin "through the mouth" or "by mou ...
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T Cell
A T cell is a type of lymphocyte. T cells are one of the important white blood cells of the immune system and play a central role in the adaptive immune response. T cells can be distinguished from other lymphocytes by the presence of a T-cell receptor (TCR) on their cell surface. T cells are born from hematopoietic stem cells, found in the bone marrow. Developing T cells then migrate to the thymus gland to develop (or mature). T cells derive their name from the thymus. After migration to the thymus, the precursor cells mature into several distinct types of T cells. T cell differentiation also continues after they have left the thymus. Groups of specific, differentiated T cell subtypes have a variety of important functions in controlling and shaping the immune response. One of these functions is immune-mediated cell death, and it is carried out by two major subtypes: CD8+ "killer" and CD4+ "helper" T cells. (These are named for the presence of the cell surface proteins CD8 or ...
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Drugs Developed By Novartis
A drug is any chemical substance that causes a change in an organism's physiology or psychology when consumed. Drugs are typically distinguished from food and substances that provide nutritional support. Consumption of drugs can be via inhalation, injection, smoking, ingestion, absorption via a patch on the skin, suppository, or dissolution under the tongue. In pharmacology, a drug is a chemical substance, typically of known structure, which, when administered to a living organism, produces a biological effect. A pharmaceutical drug, also called a medication or medicine, is a chemical substance used to treat, cure, prevent, or diagnose a disease or to promote well-being. Traditionally drugs were obtained through extraction from medicinal plants, but more recently also by organic synthesis. Pharmaceutical drugs may be used for a limited duration, or on a regular basis for chronic disorders. Pharmaceutical drugs are often classified into drug classes—groups of related drugs ...
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Antineoplastic Drugs
Chemotherapy (often abbreviated to chemo and sometimes CTX or CTx) is a type of cancer treatment that uses one or more anti-cancer drugs (chemotherapeutic agents or alkylating agents) as part of a standardized chemotherapy regimen. Chemotherapy may be given with a curative intent (which almost always involves combinations of drugs) or it may aim to prolong life or to reduce symptoms ( palliative chemotherapy). Chemotherapy is one of the major categories of the medical discipline specifically devoted to pharmacotherapy for cancer, which is called ''medical oncology''. The term ''chemotherapy'' has come to connote non-specific usage of intracellular poisons to inhibit mitosis (cell division) or induce DNA damage, which is why inhibition of DNA repair can augment chemotherapy. The connotation of the word chemotherapy excludes more selective agents that block extracellular signals ( signal transduction). The development of therapies with specific molecular or genetic targets, ...
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Microsoft
Microsoft Corporation is an American multinational technology corporation producing computer software, consumer electronics, personal computers, and related services headquartered at the Microsoft Redmond campus located in Redmond, Washington, United States. Its best-known software products are the Windows line of operating systems, the Microsoft Office suite, and the Internet Explorer and Edge web browsers. Its flagship hardware products are the Xbox video game consoles and the Microsoft Surface lineup of touchscreen personal computers. Microsoft ranked No. 21 in the 2020 Fortune 500 rankings of the largest United States corporations by total revenue; it was the world's largest software maker by revenue as of 2019. It is one of the Big Five American information technology companies, alongside Alphabet, Amazon, Apple, and Meta. Microsoft was founded by Bill Gates and Paul Allen on April 4, 1975, to develop and sell BASIC interpreters for the Altair 8800. It rose to do ...
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Bing Chat
Microsoft Copilot is a chatbot developed by Microsoft and launched on February 7, 2023. Based on a large language model, it is able to cite sources, create poems, and write both lyrics and music for songs generated by its Suno AI plugin. It is Microsoft’s primary replacement for the discontinued Cortana. The service was introduced as Bing Chat, as a built-in feature for Microsoft Bing and Microsoft Edge. Over the course of 2023, Microsoft began to unify the ''Copilot'' branding across its various chatbot products. At its Build 2023 conference, Microsoft announced its plans to integrate Copilot into Windows 11, allowing users to access it directly through the taskbar. In January 2024, a dedicated Copilot key was announced for Windows keyboards. Copilot utilizes the Microsoft Prometheus model, built upon OpenAI's GPT-4 foundational large language model, which in turn has been fine-tuned using both supervised and reinforcement learning techniques. The chatbot's conversati ...
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Priority Review
Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease. The priority review voucher program is a program that grants a voucher for priority review to a drug developer as an incentive to develop treatments for disease indications with limited profitability. Priority review vouchers are currently earned by pharmaceutical companies for the development and approval of drugs treating neglected tropical diseases, rare pediatric diseases, and "medical countermeasures" for terrorism. The voucher can be used for future drugs that could have wider indications for use, but the company is required to pay a fee (approximately $2.8 million) to use the voucher. When seeking approval for a drug, manufacturers can apply to the FDA for priority review. This is granted when a drug is intended to treat a serious condition and would "provide a si ...
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Orphan Drug
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 ...
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Biotechnology
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 ...
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Netherlands
) , anthem = ( en, "William of Nassau") , image_map = , map_caption = , subdivision_type = Sovereign state , subdivision_name = Kingdom of the Netherlands , established_title = Before independence , established_date = Spanish Netherlands , established_title2 = Act of Abjuration , established_date2 = 26 July 1581 , established_title3 = Peace of Münster , established_date3 = 30 January 1648 , established_title4 = Kingdom established , established_date4 = 16 March 1815 , established_title5 = Liberation Day (Netherlands), Liberation Day , established_date5 = 5 May 1945 , established_title6 = Charter for the Kingdom of the Netherlands, Kingdom Charter , established_date6 = 15 December 1954 , established_title7 = Dissolution of the Netherlands Antilles, Caribbean reorganisation , established_date7 = 10 October 2010 , official_languages = Dutch language, Dutch , languages_type = Regional languages , languages_sub = yes , languages = , languages2_type = Reco ...
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