CMX-001
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CMX-001
Brincidofovir, sold under the brand name Tembexa, is an antiviral drug used to treat smallpox. Brincidofovir is a prodrug of cidofovir. Conjugated to a lipid, the compound is designed to release cidofovir intracellularly, allowing for higher intracellular and lower plasma concentrations of cidofovir, effectively increasing its activity against dsDNA viruses, as well as oral bioavailability. The most common side effects include diarrhea, nausea, vomiting, and abdominal pain. Brincidofovir was approved for medical use in the United States in June 2021. Medical uses Brincidofovir is indicated for the treatment of human smallpox disease caused by variola virus. Mechanism of Action Brincidofovir is a pro-drug that is composed of cidofovir, conjugated with a lipid molecule. The lipid aspect of the molecule takes on the action of endogenous lysopghosphatidyl choline, which then is able to enter cells in the body which are infected with smallpox. Once the infected cell takes ...
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Oral Administration
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 mouth ...
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Open-label Trial
An open-label trial, or open trial, is a type of clinical trial in which information is not withheld from trial participants. In particular, both the researchers and participants know which treatment is being administered. This contrasts with a double-blinded trial, where information is withheld both from the researchers and the participants to reduce bias. Open-label trials may be appropriate for comparing two similar treatments to determine which is most effective, such as a comparison of different prescription anticoagulants, or possible relief from symptoms of some disorders when a placebo is given. An open-label trial may still be randomized. Open-label trials may also be uncontrolled (without a placebo group Clinical trials are prospective biomedical or behavioral research studies on human participants designed to answer specific questions about biomedical or behavioral interventions, including new treatments (such as novel vaccines, drugs, dieta ...), with all parti ...
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Oxford University
Oxford () is a city in England. It is the county town and only city of Oxfordshire. In 2020, its population was estimated at 151,584. It is north-west of London, south-east of Birmingham and north-east of Bristol. The city is home to the University of Oxford, the oldest university in the English-speaking world; it has buildings in every style of English architecture since late Anglo-Saxon. Oxford's industries include motor manufacturing, education, publishing, information technology and science. History The history of Oxford in England dates back to its original settlement in the Saxon period. Originally of strategic significance due to its controlling location on the upper reaches of the River Thames at its junction with the River Cherwell, the town grew in national importance during the early Norman period, and in the late 12th century became home to the fledgling University of Oxford. The city was besieged during The Anarchy in 1142. The university rose to domina ...
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Trudie Lang
Trudie Lang is a Professor of Global Health Research at the University of Oxford. She specialises in clinical trials research capacity building in low-resource setting, and helped to organise the trial for the drug brincidofovir during the 2014 Ebola virus outbreak. Education and career Lang started her career working in pharmaceutical companies, including Syntex Pharmaceuticals and Glaxosmithkline. She later worked in Kenya as head of the Kilifi Clinical Trial Facility at the KEMRI-Wellcome Trust Research Programme. Lang then moved to the Nuffield Department of Medicine, University of Oxford, where she is currently Professor of Global Health Research in the Centre for Tropical Medicine and Global Health. She was promoted to professor in 2014 and is a senior research fellow of Green Templeton College. Research Her work has focused on improving clinical trials in low and middle-income countries, including training of clinical teams, strengthening regulatory protocols, and ...
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Peter Horby
Sir Peter William Horby is a British physician, epidemiologist, Moh Family Foundation Professor of Emerging Infections and Global Health, and Director of the Pandemic Sciences Institute at the University of Oxford. He is the founder, and former director of the Oxford University Clinical Research Unit in Hanoi, Vietnam which was founded in 2006. In 2014, Horby established the Epidemic Research Group Oxford (ERGO). ERGO incorporates a number of international projects such as the European Commission funded PREPARE, the African coaLition for Epidemic Research, Response and Training (ALERRT), and the International Severe Acute Respiratory and emerging Infection Consortium (ISARIC). Since 2016, Horby has been chair and executive director of ISARIC. Horby specialises in emerging and epidemic infectious diseases. He is the co-chief investigator of the RECOVERY Trial into drugs for COVID-19 (the largest clinical trial of COVID-19 in the world) alongside Martin Landray. In June 2020, he ...
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2014 United States Ebola Virus Outbreak
Four laboratory-confirmed cases of Ebola virus disease (commonly known as "Ebola") occurred in the United States in 2014. Eleven cases were reported, including these four cases and seven cases medically evacuated from other countries. The first was reported in September 2014. Nine of the people contracted the disease outside the US and traveled into the country, either as regular airline passengers or as Medical evacuation, medical evacuees; of those nine, two died. Two people contracted Ebola in the United States. Both were nurses who treated an Ebola patient; both recovered. On September 30, 2014, the Centers for Disease Control and Prevention (CDC) announced that Thomas Eric Duncan, a reportedly 42-year-old (later corrected by CDC reports as a 45-year-old) Liberian people, Liberian national visiting the United States from Liberia, had been diagnosed with Ebola in Dallas, Texas. Duncan, who had been visiting family in Dallas, was treated at Presbyterian Hospital of Dallas, Texa ...
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Kidney Transplants
Kidney transplant or renal transplant is the organ transplant of a kidney into a patient with end-stage kidney disease (ESRD). Kidney transplant is typically classified as deceased-donor (formerly known as cadaveric) or living-donor transplantation depending on the source of the donor organ. Living-donor kidney transplants are further characterized as genetically related (living-related) or non-related (living-unrelated) transplants, depending on whether a biological relationship exists between the donor and recipient. Before receiving a kidney transplant, a person with ESRD must undergo a thorough medical evaluation to make sure that they are healthy enough to undergo transplant surgery. If they are deemed a good candidate, they can be placed on a waiting list to receive a kidney from a deceased donor. Once they are placed on the waiting list, they can receive a new kidney very quickly, or they may have to wait many years; in the United States, the average waiting time is three t ...
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Stem Cell Transplant
Hematopoietic stem-cell transplantation (HSCT) is the transplantation of multipotent hematopoietic stem cells, usually derived from bone marrow, peripheral blood, or umbilical cord blood in order to replicate inside of a patient and to produce additional normal blood cells. It may be autologous (the patient's own stem cells are used), allogeneic (the stem cells come from a donor) or syngeneic (from an identical twin). It is most often performed for patients with certain cancers of the blood or bone marrow, such as multiple myeloma or leukemia. In these cases, the recipient's immune system is usually destroyed with radiation or chemotherapy before the transplantation. Infection and graft-versus-host disease are major complications of allogeneic HSCT. HSCT remains a dangerous procedure with many possible complications; it is reserved for patients with life-threatening diseases. As survival following the procedure has increased, its use has expanded beyond cancer to autoimmun ...
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Phase III Clinical Trial
The phases of clinical research are the stages in which scientists conduct experiments with a health intervention to obtain sufficient evidence for a process considered effective as a medical treatment. For drug development, the clinical phases start with testing for safety in a few human subjects, then expand to many study participants (potentially tens of thousands) to determine if the treatment is effective. Clinical research is conducted on drug candidates, vaccine candidates, new medical devices, and new diagnostic assays. Summary Clinical trials testing potential medical products are commonly classified into four phases. The drug development process will normally proceed through all four phases over many years. If the drug successfully passes through Phases I, II, and III, it will usually be approved by the national regulatory authority for use in the general population. Phase IV trials are 'post-marketing' or 'surveillance' studies conducted to monitor safety over sever ...
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Monkeypox
Monkeypox (also called mpox by the WHO) is an infectious viral disease that can occur in humans and some other animals. Symptoms include fever, swollen lymph nodes, and a rash that forms blisters and then crusts over. The time from exposure to onset of symptoms ranges from five to twenty-one days. The duration of symptoms is typically two to four weeks. There may be mild symptoms, and it may occur without any symptoms being apparent. The classic presentation of fever and muscle pains, followed by swollen glands, with lesions all at the same stage, has not been found to be common to all outbreaks. Cases may be severe, especially in children, pregnant women or people with suppressed immune systems. The disease is caused by the monkeypox virus, a zoonotic virus in the genus ''Orthopoxvirus''. The variola virus, the causative agent of smallpox, is also in this genus. Of the two types in humans, cladeII (formerly West African clade) causes a less severe disease than the Central A ...
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Ebolavirus
The genus ''Ebolavirus'' (- or ; - or ) is a virological taxon included in the family ''Filoviridae'' (filament-shaped viruses), order ''Mononegavirales''. The members of this genus are called ebolaviruses, and encode their genome in the form of single-stranded negative-sense RNA. The six known virus species are named for the region where each was originally identified: ''Bundibugyo ebolavirus'', '' Reston ebolavirus'', '' Sudan ebolavirus'', ''Taï Forest ebolavirus'' (originally ''Côte d'Ivoire ebolavirus''), ''Zaire ebolavirus'', and ''Bombali ebolavirus''. The last is the most recent species to be named and was isolated from Angolan free-tailed bats in Sierra Leone. Each species of the genus ''Ebolavirus'' has one member virus, and four of these cause Ebola virus disease (EVD) in humans, a type of hemorrhagic fever having a very high case fatality rate. The Reston virus has caused EVD in other primates. ''Zaire ebolavirus'' has the highest mortality rate of the ebolaviru ...
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Poxvirus
''Poxviridae'' is a family of double-stranded DNA viruses. Vertebrates and arthropods serve as natural hosts. There are currently 83 species in this family, divided among 22 genera, which are divided into two subfamilies. Diseases associated with this family include smallpox. Four genera of poxviruses may infect humans: ''Orthopoxvirus'', ''Parapoxvirus'', ''Yatapoxvirus'', ''Molluscipoxvirus''. ''Orthopoxvirus'': smallpox virus (variola), vaccinia virus, cowpox virus, monkeypox virus; ''Parapoxvirus'': orf virus, pseudocowpox, bovine papular stomatitis virus; ''Yatapoxvirus'': tanapox virus, yaba monkey tumor virus; ''Molluscipoxvirus'': molluscum contagiosum virus (MCV). The most common are vaccinia (seen on the Indian subcontinent) and molluscum contagiosum, but monkeypox infections are rising (seen in west and central African rainforest countries). The similarly named disease chickenpox is not a true poxvirus and is caused by the herpesvirus varicella zoster. Etymology ...
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