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Delandistrogene Moxeparvovec
Delandistrogene moxeparvovec, sold under the brand name Elevidys, is a recombinant gene therapy used for the treatment of Duchenne muscular dystrophy. It is designed to deliver into the body a gene that leads to production of Elevidys micro-dystrophin that contains selected domains of the dystrophin protein present in normal muscle cells. It is an adeno-associated virus vector-based gene therapy that is given by intravenous infusion (slow injection into a vein). The most commonly reported side effects include vomiting, nausea, acute liver injury, pyrexia (fever), and thrombocytopenia (abnormally low platelet count in the blood). One patient may have died from acute liver failure. Delandistrogene moxeparvovec was approved for medical use in the United States in June 2023. It was developed by Sarepta Therapeutics, together with Roche, and is manufactured by Catalent. Medical uses Delandistrogene moxeparvovec is indicated In medicine, an indication is a valid reason to us ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Intravenous Infusion
Intravenous therapy (abbreviated as IV therapy) is a medical technique that administers fluids, medications and nutrients directly into a person's vein. The intravenous route of administration is commonly used for rehydration or to provide nutrients for those who cannot, or will not—due to reduced mental states or otherwise—consume food or water by mouth. It may also be used to administer medications or other medical therapy such as blood products or electrolytes to correct electrolyte imbalances. Attempts at providing intravenous therapy have been recorded as early as the 1400s, but the practice did not become widespread until the 1900s after the development of techniques for safe, effective use. The intravenous route is the fastest way to deliver medications and fluid replacement throughout the body as they are introduced directly into the circulatory system and thus quickly distributed. For this reason, the intravenous route of administration is also used for the consumpt ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Sarepta Therapeutics
Sarepta Therapeutics, Inc. is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States. Incorporated in 1980 as AntiVirals, , archiveurl = http://www.gene-tools.com/sites/default/files/Summerton2005_IPT.pdf shortly before going public the company changed its name from AntiVirals to AVI BioPharma soon with stock symbol AVII and in July 2012 changed name from AVI BioPharma to Sarepta Therapeutics and SRPT respectively. As of 2023, the company has four approved drugs (see the Products section below). History Sarepta started in Corvallis, Oregon on January 1, 1980 and was originally named Antivirals Inc. After occupying several research laboratory spaces in Corvallis, the company opened a production laboratory in Corvallis in February 2002 and was renamed AVI BioPharma Inc. The company made headlines in 2003 when it announced work on treatments for severe acute respiratory syndrome (SARS) and the West N ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Medical Treatments
Medicine is the science and Praxis (process), practice of caring for patients, managing the Medical diagnosis, diagnosis, prognosis, Preventive medicine, prevention, therapy, treatment, Palliative care, palliation of their injury or disease, and Health promotion, promoting their health. Medicine encompasses a variety of health care practices evolved to maintain and restore health by the prevention (medical), prevention and treatment of illness. Contemporary medicine applies biomedical sciences, biomedical research, medical genetics, genetics, and medical technology to diagnosis (medical), diagnose, treat, and prevent injury and disease, typically through pharmaceuticals or surgery, but also through therapies as diverse as psychotherapy, splint (medicine), external splints and traction, medical devices, biologic medical product, biologics, and Radiation (medicine), ionizing radiation, amongst others. Medicine has been practiced since Prehistoric medicine, prehistoric times, and ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Approved Gene Therapies
Approved may refer to: *Approved drug, a preparation that has been validated for a therapeutic use by a ruling authority of a government *''Approved'', a 2013 album by Chester Thompson Chester Thompson (born December 11, 1948) is an American drummer best known for his tenures with Frank Zappa and the Mothers of Invention, Weather Report, Santana (band), Santana, Genesis (band), Genesis and Phil Collins. Thompson has performed ... Trio * ''Approved'' (Ubiquitous Synergy Seeker album) {{disambiguation ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Biotechnology
Biotechnology is a multidisciplinary field that involves the integration of natural sciences and Engineering Science, engineering sciences in order to achieve the application of organisms and parts thereof for products and services. Specialists in the field are known as biotechnologists. The term ''biotechnology'' was first used by Károly Ereky in 1919 to refer to the production of products from raw materials with the aid of living organisms. The core principle of biotechnology involves harnessing biological systems and organisms, such as bacteria, yeast, and plants, to perform specific tasks or produce valuable substances. Biotechnology had a significant impact on many areas of society, from medicine to agriculture to environmental science. One of the key techniques used in biotechnology is genetic engineering, which allows scientists to modify the genetic makeup of organisms to achieve desired outcomes. This can involve inserting genes from one organism into another, and con ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
The Boston Globe
''The Boston Globe,'' also known locally as ''the Globe'', is an American daily newspaper founded and based in Boston, Massachusetts. The newspaper has won a total of 27 Pulitzer Prizes. ''The Boston Globe'' is the oldest and largest daily newspaper in Boston and tenth-largest newspaper by print circulation in the nation as of 2023. Founded in 1872, the paper was mainly controlled by Irish Catholic interests before being sold to Charles H. Taylor and his family. After being privately held until 1973, it was sold to ''The New York Times'' in 1993 for $1.1billion, making it one of the most expensive print purchases in United States history. The newspaper was purchased in 2013 by Boston Red Sox and Liverpool F.C. owner John W. Henry for $70million from The New York Times Company, having lost over 90% of its value in 20 years. The chief print rival of ''The Boston Globe'' is the '' Boston Herald'', whose circulation is smaller and is shrinking faster. The newspaper is "one ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Indicated
In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery. There can be multiple indications to use a procedure or medication. An indication can commonly be confused with the term diagnosis. A diagnosis is the assessment that a particular medical condition is present while an indication is a reason for use. The opposite of an indication is a contraindication, a reason to withhold a certain medical treatment because the risks of treatment clearly outweigh the benefits. In the United States, indications for prescription drugs are approved by the FDA. Indications are included in the Indications and Usage section of the Prescribing Information. The primary role of this section of labeling is to enable health care practitioners to readily identify appropriate therapies for patients by clearly communicating the drug's approved indication(s). The Indications and Usage section states the disease or condition, or manifestation or symptoms thereof ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Catalent
Catalent, Inc. (Catalent Pharma Solutions), is an American multinational company, and a subsidiary of Novo Holdings A/S since 2024. It is headquartered in Somerset, New Jersey. It is a provider of drug delivery technologies, drug development, drug manufacturing, biologics, gene therapy, and consumer health products. It has over 50 facilities on four continents and has supported more than half the products approved by the Food and Drug Administration in the last ten years. Annually, it produces 70 billion doses for 8,000 products. The company's major customers include Bayer, Bristol-Myers Squibb, GlaxoSmithKline, Haleon, Novo Nordisk, Moderna, Pfizer, and Sarepta Therapeutics. History Before 2007 In 1996, Cardinal Health acquired PCI of Philadelphia, Pennsylvania, a pharmaceutical contract packing service for commercial and clinical packaging. In 1998, Cardinal Health acquired R.P. Scherer Corporation of Troy, Michigan for $2.2 billion; it was founded by Robert Pauli Sche ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Roche
F. Hoffmann-La Roche AG, commonly known as Roche (), is a Switzerland, Swiss multinational corporation, multinational holding healthcare company that operates worldwide under two divisions: Pharmaceuticals and Diagnostics. Its holding company, Roche Holding AG, has Share (finance), shares listed on the SIX Swiss Exchange. The company headquarters are located in Basel. Roche is the fifth-largest pharmaceutical company in the world by revenue and the leading provider of cancer treatments globally. In 2023, the company’s seat in Forbes Global 2000 was 76. The company owns the American biotechnology company Genentech, which is a wholly owned independent subsidiary, and the Japanese biotechnology company Chugai Pharmaceutical Co., Chugai Pharmaceuticals, as well as the United States–based companies Ventana Medical Systems, Ventana and Foundation Medicine. Roche's revenues during fiscal year 2020, were 58.32 billion Swiss francs. Descendants of the founding Hoffmann and Oeri fam ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Thrombocytopenia
In hematology, thrombocytopenia is a condition characterized by abnormally low levels of platelets (also known as thrombocytes) in the blood. Low levels of platelets in turn may lead to prolonged or excessive bleeding. It is the most common coagulation disorder among intensive care units, intensive care patients and is seen in a fifth of medical patients and a third of surgical patients. A normal human platelet count ranges from 150,000 to 450,000 platelets/microliter (μL) of blood. Values outside this range do not necessarily indicate disease. One common definition of thrombocytopenia requiring emergency treatment is a platelet count below 50,000/μL. Thrombocytopenia can be contrasted with the conditions associated with an abnormally ''high'' level of platelets in the blood – thrombocythemia (when the cause is unknown), and thrombocytosis (when the cause is known). Signs and symptoms Thrombocytopenia usually asymptomatic, has no symptoms and is picked up on a routine comp ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Food And Drug Administration
The United States Food and Drug Administration (FDA or US FDA) is a List of United States federal agencies, federal agency of the United States Department of Health and Human Services, Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, Prescription drug, prescription and Over-the-counter drug, over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, Animal feed, animal foods & feed and Veterinary medicine, veterinary products. The FDA's primary focus is enforcement of the Federal Food, Drug, and Cosmetic Act (FD&C). However, the agency also enforces other laws, notably Section 361 of the Public Health Service Act as well as associated regulations. Much of this regulatory-enforcement work is ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Fever
Fever or pyrexia in humans is a symptom of an anti-infection defense mechanism that appears with Human body temperature, body temperature exceeding the normal range caused by an increase in the body's temperature Human body temperature#Fever, set point in the hypothalamus. There is no single agreed-upon upper limit for normal temperature: sources use values ranging between in humans. The increase in set point triggers increased muscle tone, muscle contractions and causes a feeling of cold or chills. This results in greater heat production and efforts to conserve heat. When the set point temperature returns to normal, a person feels hot, becomes Flushing (physiology), flushed, and may begin to Perspiration, sweat. Rarely a fever may trigger a febrile seizure, with this being more common in young children. Fevers do not typically go higher than . A fever can be caused by many medical conditions ranging from non-serious to life-threatening. This includes viral infection, viral, b ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
