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C. Frank Bennett
C. Frank Bennett is an American pharmacologist. Bennett is currently the Senior Vice President of Research and Neurology Franchise Leader at Ionis Pharmaceuticals. He is a 2019 Breakthrough Prize winner in Life Sciences, which he shared with his collaborator Adrian R. Krainer for the development of an effective antisense oligonucleotide therapy for children with the neurodegenerative disease spinal muscular atrophy. Early life and education C. Frank Bennett grew up in Aztec, New Mexico, where his family owned a small hotel. Bennett holds a bachelor's degree in Pharmacy from the University of New Mexico and obtained a PhD in Pharmacology in 1985 from Baylor College of Medicine. Bennett completed a postdoctoral fellowship at SmithKline & French Laboratories with Stanley T. Crooke. Career Bennett is one of the founding members of Ionis Pharmaceuticals in Carlsbad, California. He has been involved in the development of antisense oligonucleotides as therapeutic agents, including ...
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Ionis Pharmaceuticals
Ionis Pharmaceuticals, Inc. is a biotechnology company based in Carlsbad, California, that specializes in discovering and developing RNA-targeted therapeutics. The company has 3 commercially approved medicines: Spinraza ( Nusinersen), Tegsedi ( Inotersen), and Waylivra (Volanesorsen) and has 4 drugs in pivotal studies: tominersen for Huntington’s disease, tofersen for SOD1- ALS, AKCEA-APO(a)-LRx for cardiovascular disease, and AKCEA-TTR-LRx for all forms of TTR amyloidosis. The company was named Isis Pharmaceuticals until December 2015. History The company was founded in 1989 as Isis Pharmaceuticals by Stanley T. Crooke, a former head of research of GlaxoSmithKline, with a goal to commercialize antisense therapy. In 1992, the company received its first approval by the Food and Drug Administration for an investigational new drug application in 1992 for a genital warts drug candidate. The FDA approval marked the first time for the company to conduct any antisense therapy and ...
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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. Anoth ...
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21st-century American Biologists
The 1st century was the century spanning AD 1 (Roman numerals, I) through AD 100 (Roman numerals, C) according to the Julian calendar. It is often written as the or to distinguish it from the 1st century BC (or BCE) which preceded it. The 1st century is considered part of the Classical era, epoch, or History by period, historical period. The 1st century also saw the Christianity in the 1st century, appearance of Christianity. During this period, Europe, North Africa and the Near East fell under increasing domination by the Roman Empire, which continued expanding, most notably conquering Britain under the emperor Claudius (AD 43). The reforms introduced by Augustus during his long reign stabilized the empire after the turmoil of the previous century's civil wars. Later in the century the Julio-Claudian dynasty, which had been founded by Augustus, came to an end with the suicide of Nero in AD 68. There followed the famous Year of Four Emperors, a brief period of civil war and inst ...
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1960 Births
Year 196 ( CXCVI) was a leap year starting on Thursday (link will display the full calendar) of the Julian calendar. At the time, it was known as the Year of the Consulship of Dexter and Messalla (or, less frequently, year 949 ''Ab urbe condita''). The denomination 196 for this year has been used since the early medieval period, when the Anno Domini calendar era became the prevalent method in Europe for naming years. Events By place Roman Empire * Emperor Septimius Severus attempts to assassinate Clodius Albinus but fails, causing Albinus to retaliate militarily. * Emperor Septimius Severus captures and sacks Byzantium; the city is rebuilt and regains its previous prosperity. * In order to assure the support of the Roman legion in Germany on his march to Rome, Clodius Albinus is declared Augustus by his army while crossing Gaul. * Hadrian's wall in Britain is partially destroyed. China * First year of the '' Jian'an era of the Chinese Han Dynasty. * Em ...
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Hereditary Disease Foundation
The Hereditary Disease Foundation (HDF) aims to cure genetic disorders, notably Huntington's disease, by supporting basic biomedical research. History In 1968, after experiencing Huntington's disease (HD) in his wife's family, Milton Wexler was inspired to start the Hereditary Disease Foundation, with the aim of curing genetic illnesses by co-ordinating and supporting research. At a workshop held by the HDF in 1979, participants proposed to map the human genome and find a marker for the gene which causes HD. The HDF, together with the National Institute of Neurological Disorders and Stroke and Wexler's daughter, Nancy Wexler, organized the US–Venezuela Huntington's Disease Collaborative Research Project. This project studied a kindred with an unusually high prevalence of HD. In 1983, a marker for a gene was found, and in the next decade, with further HDF involvement, the exact gene (Huntingtin) was found. Many techniques developed in finding the Huntingtin gene were used to ad ...
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Amyotrophic Lateral Sclerosis
Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND) or Lou Gehrig's disease, is a neurodegenerative disease that results in the progressive loss of motor neurons that control voluntary muscles. ALS is the most common type of motor neuron diseases. Early symptoms of ALS include stiff muscles, muscle twitches, and gradual increasing weakness and muscle wasting. ''Limb-onset ALS'' begins with weakness in the arms or legs, while ''bulbar-onset ALS'' begins with difficulty speaking or swallowing. Half of the people with ALS develop at least mild difficulties with thinking and behavior, and about 15% develop frontotemporal dementia. Most people experience pain. The affected muscles are responsible for chewing food, speaking, and walking. Motor neuron loss continues until the ability to eat, speak, move, and finally the ability to breathe is lost. ALS eventually causes paralysis and early death, usually from respiratory failure. Most cases of ALS (a ...
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Alzheimer's Disease
Alzheimer's disease (AD) is a neurodegeneration, neurodegenerative disease that usually starts slowly and progressively worsens. It is the cause of 60–70% of cases of dementia. The most common early symptom is difficulty in short-term memory, remembering recent events. As the disease advances, symptoms can include primary progressive aphasia, problems with language, Orientation (mental), disorientation (including easily getting lost), mood swings, loss of motivation, self-neglect, and challenging behaviour, behavioral issues. As a person's condition declines, they often withdraw from family and society. Gradually, bodily functions are lost, ultimately leading to death. Although the speed of progression can vary, the typical life expectancy following diagnosis is three to nine years. The cause of Alzheimer's disease is poorly understood. There are many environmental and genetic risk factors associated with its development. The strongest genetic risk factor is from an alle ...
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Huntington's Disease
Huntington's disease (HD), also known as Huntington's chorea, is a neurodegenerative disease that is mostly inherited. The earliest symptoms are often subtle problems with mood or mental abilities. A general lack of coordination and an unsteady gait often follow. It is also a basal ganglia disease causing a hyperkinetic movement disorder known as chorea. As the disease advances, uncoordinated, involuntary body movements of chorea become more apparent. Physical abilities gradually worsen until coordinated movement becomes difficult and the person is unable to talk. Mental abilities generally decline into dementia. The specific symptoms vary somewhat between people. Symptoms usually begin between 30 and 50 years of age but can start at any age. The disease may develop earlier in each successive generation. About eight percent of cases start before the age of 20 years, and are known as ''juvenile HD'', which typically present with the slow movement symptoms of Parkinso ...
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Nusinersen
Nusinersen, marketed as Spinraza, is a medication used in treating spinal muscular atrophy (SMA), a rare neuromuscular disorder. In December 2016, it became the first approved drug used in treating this disorder. Since the condition it treats is so rare, Nusinersen has so-called "orphan drug" designation in the United States and the European Union. Medical uses The drug is used to treat spinal muscular atrophy associated with a mutation in the '' SMN1'' gene. It is administered directly to the central nervous system (CNS) using intrathecal injection. In clinical trials, the drug halted the disease progression. In around 60% of infants affected by type 1 spinal muscular atrophy, it improves motor function. Side effects People treated with nusinersen had an increased risk of upper and lower respiratory infections and congestion, ear infections, constipation, pulmonary aspiration, teething, and scoliosis. There is a risk that growth of infants and children might be stunted. In o ...
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University Of New Mexico
The University of New Mexico (UNM; es, Universidad de Nuevo México) is a public research university in Albuquerque, New Mexico. Founded in 1889, it is the state's flagship academic institution and the largest by enrollment, with over 25,400 students in 2021. UNM comprises twelve colleges and schools, including the only law school in New Mexico. It offers 94 baccalaureate, 71 masters, and 37 doctoral degrees. The main campus spans in central Albuquerque, with branch campuses in Gallup, Los Alamos, Rio Rancho, Taos, and Los Lunas. UNM is classified among "R1: Doctoral Universities – Very high research activity", and spent over $243 million on research and development in 2021, ranking 103rd in the nation. UNM's NCAA Division I program ( FBS for football) offers 16 varsity sports; known as the Lobos, the teams compete in the Mountain West Conference and have won national championships in skiing and cross country running. The official school colors are cherry ...
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Antisense Oligonucleotide
Oligonucleotides are short DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, and forensics. Commonly made in the laboratory by solid-phase chemical synthesis, these small bits of nucleic acids can be manufactured as single-stranded molecules with any user-specified sequence, and so are vital for artificial gene synthesis, polymerase chain reaction (PCR), DNA sequencing, molecular cloning and as molecular probes. In nature, oligonucleotides are usually found as small RNA molecules that function in the regulation of gene expression (e.g. microRNA), or are degradation intermediates derived from the breakdown of larger nucleic acid molecules. Oligonucleotides are characterized by the sequence of nucleotide residues that make up the entire molecule. The length of the oligonucleotide is usually denoted by "-mer" (from Greek ''meros'', "part"). For example, an oligonucleotide of six nucleotides (nt) is a hexamer, while one of 25 nt wo ...
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Carlsbad, California
Carlsbad is a coastal city in the North County region of San Diego County, California, United States. The city is south of downtown Los Angeles and north of downtown San Diego. As of the 2020 census, the population of the city was 114,746. Carlsbad is a popular tourist destination and home to many businesses in the golf industry. History Carlsbad's history began with the Luiseño people (the Spanish name given to them because of their proximity to Mission San Luis Rey), as well as some Kumeyaay in the La Costa area. Nearly every reliable fresh water creek had at least one native village, including one called Palamai. The site is located just south of today's Buena Vista Lagoon. The first European land exploration of Alta California, the Spanish Portolá expedition of 1769, met native villagers while camped on Buena Vista Creek. Another Luiseño villages within today's city of Carlsbad was a village at the mouth of the San Marcos Creek that the Kumeyaay called 'Ajopun ...
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