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Golodirsen
Golodirsen, sold under the brand name Vyondys 53, is a medication used for the treatment of Duchenne muscular dystrophy (DMD). It is an antisense oligonucleotide drug of phosphorodiamidate morpholino oligomer (PMO) chemistry. The most common side effects include headache, fever, fall, cough, vomiting, abdominal pain, cold symptoms ( nasopharyngitis) and nausea. Medical uses Golodirsen is indicated for the treatment of Duchenne muscular dystrophy (DMD) in people who have a confirmed mutation of the ''dystrophin'' gene that is amenable to exon 53 skipping. Mechanism of action Golodirsen has been provisionally approved for approximately 8% of all DMD patients amenable to exon 53 skipping. It works by inducing exon skipping in the dystrophin gene and thereby increasing the amount of dystrophin protein available to muscle fibers. Adverse effects The most common side effects include headache, fever, fall, cough, vomiting, abdominal pain, cold symptoms ( nasopharyngitis) and nau ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Exon Skipping
In molecular biology, exon skipping is a form of RNA splicing used to cause cells to “skip” over faulty or misaligned sections (exons) of genetic code, leading to a truncated but still functional protein despite the genetic mutation. Mechanism Exon skipping is used to restore the reading frame within a gene. Genes are the genetic instructions for creating a protein, and are composed of introns and exons. Exons are the sections of DNA that contain the instruction set for generating a protein; they are interspersed with non-coding regions called introns. The introns are later removed before the protein is made, leaving only the coding exon regions. Splicing naturally occurs in pre-mRNA when introns are being removed to form mature-mRNA that consists solely of exons. Starting in the late 1990s, scientists realized they could take advantage of this naturally occurring cellular splicing to downplay genetic mutations into less harmful ones. The mechanism behind exon skipping is a ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Intravenous Therapy
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 cons ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Mutation
In biology, a mutation is an alteration in the nucleic acid sequence of the genome of an organism, virus, or extrachromosomal DNA. Viral genomes contain either DNA or RNA. Mutations result from errors during DNA or viral replication, mitosis, or meiosis or other types of damage to DNA (such as pyrimidine dimers caused by exposure to ultraviolet radiation), which then may undergo error-prone repair (especially microhomology-mediated end joining), cause an error during other forms of repair, or cause an error during replication ( translesion synthesis). Mutations may also result from insertion or deletion of segments of DNA due to mobile genetic elements. Mutations may or may not produce detectable changes in the observable characteristics ( phenotype) of an organism. Mutations play a part in both normal and abnormal biological processes including: evolution, cancer, and the development of the immune system, including junctional diversity. Mutation is the ultima ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Sprain
A sprain, also known as a torn ligament, is an acute soft tissue injury of the ligaments within a joint, often caused by a sudden movement abruptly forcing the joint to exceed its functional range of motion. Ligaments are tough, inelastic fibers made of collagen that connect two or more bones to form a joint and are important for joint stability and proprioception, which is the body's sense of limb position and movement. Sprains can occur at any joint but most commonly occur in the ankle, knee, or wrist. An equivalent injury to a muscle or tendon is known as a strain. The majority of sprains are mild, causing minor swelling and bruising that can be resolved with conservative treatment, typically summarized as RICE: rest, ice, compression, elevation. However, severe sprains involve complete tears, ruptures, or fractures, often leading to joint instability, severe pain, and decreased functional ability. These sprains require surgical fixation, prolonged immobilization, and ph ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Oropharyngeal
The pharynx (plural: pharynges) is the part of the throat behind the mouth and nasal cavity, and above the oesophagus and trachea (the tubes going down to the stomach and the lungs). It is found in vertebrates and invertebrates, though its structure varies across species. The pharynx carries food and air to the esophagus and larynx respectively. The flap of cartilage called the epiglottis stops food from entering the larynx. In humans, the pharynx is part of the digestive system and the conducting zone of the respiratory system. (The conducting zone—which also includes the nostrils of the nose, the larynx, trachea, bronchi, and bronchioles—filters, warms and moistens air and conducts it into the lungs). The human pharynx is conventionally divided into three sections: the nasopharynx, oropharynx, and laryngopharynx. It is also important in vocalization. In humans, two sets of pharyngeal muscles form the pharynx and determine the shape of its lumen. They are arranged as ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Back Pain
Back pain is pain Pain is a distressing feeling often caused by intense or damaging stimuli. The International Association for the Study of Pain defines pain as "an unpleasant sensory and emotional experience associated with, or resembling that associated with, ... felt in the back. It may be classified as neck pain (cervical), middle back pain (thoracic), lower back pain (lumbar) or coccydynia (tailbone or sacral pain) based on the segment affected. The lumbar area is the most common area affected.Church E, Odle TDiagnosis and treatment of back pain. ''Radiologic Technology''&nbsserial online November 2007;79(2):126–204. Available from: CINAHL Plus with Full Text, Ipswich, MA. Accessed December 12, 2017. An episode of back pain may be acute, subacute or chronic depending on the duration. The pain may be characterized as a dull ache, shooting or piercing pain or a burning sensation. Discomfort can radiate to the arms and hands as well as the legs or feet, an ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Intravenous
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 consu ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Pain
Pain is a distressing feeling often caused by intense or damaging stimuli. The International Association for the Study of Pain defines pain as "an unpleasant sensory and emotional experience associated with, or resembling that associated with, actual or potential tissue damage." In medical diagnosis, pain is regarded as a symptom of an underlying condition. Pain motivates the individual to withdraw from damaging situations, to protect a damaged body part while it heals, and to avoid similar experiences in the future. Most pain resolves once the noxious stimulus is removed and the body has healed, but it may persist despite removal of the stimulus and apparent healing of the body. Sometimes pain arises in the absence of any detectable stimulus, damage or disease. Pain is the most common reason for physician consultation in most developed countries. It is a major symptom in many medical conditions, and can interfere with a person's quality of life and general functioning. Simp ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Clinical Trials
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, dietary choices, dietary supplements, and medical devices) and known interventions that warrant further study and comparison. Clinical trials generate data on dosage, safety and efficacy. They are conducted only after they have received health authority/ethics committee approval in the country where approval of the therapy is sought. These authorities are responsible for vetting the risk/benefit ratio of the trial—their approval does not mean the therapy is 'safe' or effective, only that the trial may be conducted. Depending on product type and development stage, investigators initially enroll volunteers or patients into small pilot studies, and subsequently conduct progressively larger scale comparative studies. Clinical trials can vary i ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Subcutaneous Injection
Subcutaneous administration is the insertion of medications beneath the skin either by injection or infusion. A subcutaneous injection is administered as a bolus into the subcutis, the layer of skin directly below the dermis and epidermis, collectively referred to as the cutis. The instruments are usually a hypodermic needle and a syringe. Subcutaneous injections are highly effective in administering medications such as insulin, morphine, diacetylmorphine and goserelin. Subcutaneous administration may be abbreviated as SC, SQ, subcu, sub-Q, SubQ, or subcut. Subcut is the preferred abbreviation to reduce the risk of misunderstanding and potential errors. Subcutaneous tissue has few blood vessels and so drugs injected here are for slow, sustained rates of absorption, often with some amount of depot effect. Compared with other routes of administration, it is slower than intramuscular injections but still faster than intradermal injections. Subcutaneous infusion (as opp ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Mice
A mouse ( : mice) is a small rodent. Characteristically, mice are known to have a pointed snout, small rounded ears, a body-length scaly tail, and a high breeding rate. The best known mouse species is the common house mouse (''Mus musculus''). Mice are also popular as pets. In some places, certain kinds of field mice are locally common. They are known to invade homes for food and shelter. Mice are typically distinguished from rats by their size. Generally, when a muroid rodent is discovered, its common name includes the term ''mouse'' if it is smaller, or ''rat'' if it is larger. The common terms ''rat'' and ''mouse'' are not taxonomically specific. Typical mice are classified in the genus '' Mus'', but the term ''mouse'' is not confined to members of ''Mus'' and can also apply to species from other genera such as the deer mouse, ''Peromyscus''. Domestic mice sold as pets often differ substantially in size from the common house mouse. This is attributable to breeding a ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Monkeys
Monkey is a common name that may refer to most mammals of the infraorder Simiiformes, also known as the simians. Traditionally, all animals in the group now known as simians are counted as monkeys except the apes, which constitutes an incomplete Paraphyly, paraphyletic grouping; however, in the broader sense based on cladistics, apes (Hominoidea) are also included, making the terms ''monkeys'' and ''simians'' synonyms in regards to their scope. In 1812, Étienne Geoffroy Saint-Hilaire, Geoffroy grouped the Ape, apes and the Cercopithecidae group of monkeys together and established the name Catarrhini, "Old World monkeys", ("''singes de l'Ancien Monde''" in French language, French). The extant sister of the Catarrhini in the monkey ("singes") group is the Platyrrhini (New World monkeys). Some nine million years before the divergence between the Cercopithecidae and the apes, the Platyrrhini emerged within "monkeys" by migration to South America likely by ocean. Apes are thus de ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
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