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Risdiplam
Risdiplam, sold under the brand name Evrysdi, is a medication used to treat spinal muscular atrophy (SMA) and the first oral medication approved to treat this disease. Risdiplam is a survival of motor neuron 2-directed RNA splicing modifier. In clinical trials, the most common adverse events included fever, diarrhea, rash, ulcers of the mouth area, joint pain (arthralgia) and urinary tract infections. Additional adverse events observed in the infantile-onset population included upper respiratory tract infection, pneumonia, constipation and vomiting. Risdiplam was approved by the US Food and Drug Administration (FDA) in August 2020, for the treatment of adults and children two months of age or older. Developed in association with PTC Therapeutics and the SMA Foundation, it is marketed in the US by Genentech, a subsidiary of Roche. Medical uses In the United States, risdiplam is indicated to treat people two months of age and older with spinal muscular atrophy. Adverse ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Spinal Muscular Atrophy
Spinal muscular atrophy (SMA) is a rare neuromuscular disorder that results in the loss of motor neurons and progressive muscle wasting. It is usually diagnosed in infancy or early childhood and if left untreated it is the most common genetic cause of infant death. It may also appear later in life and then have a milder course of the disease. The common feature is progressive weakness of voluntary muscles, with arm, leg and respiratory muscles being affected first. Associated problems may include poor head control, difficulties swallowing, scoliosis, and joint contractures. The age of onset and the severity of symptoms form the basis of the traditional classification of spinal muscular atrophy into a number of types. Spinal muscular atrophy is due to an abnormality (mutation) in the ''SMN1'' gene which encodes SMN, a protein necessary for survival of motor neurons. Loss of these neurons in the spinal cord prevents signalling between the brain and skeletal muscles. Another g ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
PTC Therapeutics
PTC Therapeutics is a US pharmaceutical company focused on the development of orally administered small molecule drugs and gene therapy which regulate gene expression by targeting post-transcriptional control (PTC) mechanisms in orphan diseases. In September 2009, PTC entered into an agreement with Roche for the development of orally bioavailable small molecules for central nervous system diseases. In 2020PTC announced the FDA approval of Evrysdi™(risdiplam) for the treatment of spinal muscular atrophy (SMA) in adults and children 2 months and older, in partnership with the SMA Foundation and Roche. PTC acquired the Bio-e platform in 2019The Bio-e platformutilizes expertise in electron-transfer chemistry to modulate key biological processes beyond the reach of current drug development approaches. The lead compounds from the Bio-e platform, PTC743 and PTC857, target the enzyme 15-lipoxygenase – a key enzymatic hub that regulates the inflammation and oxidative stress that un ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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 ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Massachusetts
Massachusetts (Massachusett language, Massachusett: ''Muhsachuweesut [Massachusett writing systems, məhswatʃəwiːsət],'' English: , ), officially the Commonwealth of Massachusetts, is the most populous U.S. state, state in the New England region of the Northeastern United States. It borders on the Atlantic Ocean and Gulf of Maine to the east, Connecticut and Rhode Island to the south, New Hampshire and Vermont to the north, and New York (state), New York to the west. The state's capital and List of municipalities in Massachusetts, most populous city, as well as its cultural and financial center, is Boston. Massachusetts is also home to the urban area, urban core of Greater Boston, the largest metropolitan area in New England and a region profoundly influential upon American History of the United States, history, academia, and the Economy of the United States, research economy. Originally dependent on agriculture, fishing, and trade. Massachusetts was transformed into a manuf ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Boston
Boston (), officially the City of Boston, is the state capital and most populous city of the Commonwealth of Massachusetts, as well as the cultural and financial center of the New England region of the United States. It is the 24th- most populous city in the country. The city boundaries encompass an area of about and a population of 675,647 as of 2020. It is the seat of Suffolk County (although the county government was disbanded on July 1, 1999). The city is the economic and cultural anchor of a substantially larger metropolitan area known as Greater Boston, a metropolitan statistical area (MSA) home to a census-estimated 4.8 million people in 2016 and ranking as the tenth-largest MSA in the country. A broader combined statistical area (CSA), generally corresponding to the commuting area and including Providence, Rhode Island, is home to approximately 8.2 million people, making it the sixth most populous in the United States. Boston is one of the oldest ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Oxford University Press
Oxford University Press (OUP) is the university press of the University of Oxford. It is the largest university press in the world, and its printing history dates back to the 1480s. Having been officially granted the legal right to print books by decree in 1586, it is the second oldest university press after Cambridge University Press. It is a department of the University of Oxford and is governed by a group of 15 academics known as the Delegates of the Press, who are appointed by the vice-chancellor of the University of Oxford. The Delegates of the Press are led by the Secretary to the Delegates, who serves as OUP's chief executive and as its major representative on other university bodies. Oxford University Press has had a similar governance structure since the 17th century. The press is located on Walton Street, Oxford, opposite Somerville College, in the inner suburb of Jericho. For the last 500 years, OUP has primarily focused on the publication of pedagogical texts and ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Oxford
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 dom ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Messenger RNA
In molecular biology, messenger ribonucleic acid (mRNA) is a single-stranded molecule of RNA that corresponds to the genetic sequence of a gene, and is read by a ribosome in the process of synthesizing a protein. mRNA is created during the process of transcription, where an enzyme (RNA polymerase) converts the gene into primary transcript mRNA (also known as pre-mRNA). This pre-mRNA usually still contains introns, regions that will not go on to code for the final amino acid sequence. These are removed in the process of RNA splicing, leaving only exons, regions that will encode the protein. This exon sequence constitutes mature mRNA. Mature mRNA is then read by the ribosome, and, utilising amino acids carried by transfer RNA (tRNA), the ribosome creates the protein. This process is known as translation. All of these processes form part of the central dogma of molecular biology, which describes the flow of genetic information in a biological system. As in DNA, genetic inf ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Alternative Splicing
Alternative splicing, or alternative RNA splicing, or differential splicing, is an alternative splicing process during gene expression that allows a single gene to code for multiple proteins. In this process, particular exons of a gene may be included within or excluded from the final, processed messenger RNA (mRNA) produced from that gene. This means the exons are joined in different combinations, leading to different (alternative) mRNA strands. Consequently, the proteins translated from alternatively spliced mRNAs will contain differences in their amino acid sequence and, often, in their biological functions (see Figure). Biologically relevant alternative splicing occurs as a normal phenomenon in eukaryotes, where it increases the number of proteins that can be encoded by the genome. In humans, it is widely believed that ~95% of multi-exonic genes are alternatively spliced to produce functional alternative products from the same gene but many scientists believe that most o ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Pyridazine
Pyridazine is an aromatic, heterocyclic, organic compound with the molecular formula . It contains a six-membered ring with two adjacent nitrogen atoms. It is a colorless liquid with a boiling point of 208 °C. It is isomeric with two other diazine () rings, pyrimidine and pyrazine. Occurrence Pyridazines are rare in nature, possibly reflecting the scarcity of naturally occurring hydrazines, common building blocks for the synthesis of these heterocycles. The pyridazine structure is a popular pharmacophore which is found within a number of herbicides such as credazine, pyridafol and pyridate. It is also found within the structure of several drugs such as cefozopran, cadralazine, minaprine, pipofezine, and hydralazine. Synthesis In the course of his classic investigation on the Fischer indole synthesis, Emil Fischer prepared the first pyridazine via the condensation of phenylhydrazine and levulinic acid. The parent heterocycle was first prepared by oxidation of benzocinnol ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Taylor & Francis
Taylor & Francis Group is an international company originating in England that publishes books and academic journals. Its parts include Taylor & Francis, Routledge, F1000 (publisher), F1000 Research or Dovepress. It is a division of Informa, Informa plc, a United Kingdom–based publisher and conference company. Overview The company was founded in 1852 when William Francis (chemist), William Francis joined Richard Taylor (editor), Richard Taylor in his publishing business. Taylor had founded his company in 1798. Their subjects covered agriculture, chemistry, education, engineering, geography, law, mathematics, medicine, and social sciences. Francis's son, Richard Taunton Francis (1883–1930), was sole partner in the firm from 1917 to 1930. In 1965, Taylor & Francis launched Wykeham Publications and began book publishing. T&F acquired Hemisphere Publishing in 1988, and the company was renamed Taylor & Francis Group to reflect the growing number of Imprint (trade name), imp ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
England
England is a country that is part of the United Kingdom. It shares land borders with Wales to its west and Scotland to its north. The Irish Sea lies northwest and the Celtic Sea to the southwest. It is separated from continental Europe by the North Sea to the east and the English Channel to the south. The country covers five-eighths of the island of Great Britain, which lies in the North Atlantic, and includes over 100 smaller islands, such as the Isles of Scilly and the Isle of Wight. The area now called England was first inhabited by modern humans during the Upper Paleolithic period, but takes its name from the Angles, a Germanic tribe deriving its name from the Anglia peninsula, who settled during the 5th and 6th centuries. England became a unified state in the 10th century and has had a significant cultural and legal impact on the wider world since the Age of Discovery, which began during the 15th century. The English language, the Anglican Church, and Engli ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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