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Golodirsen, the Glossary

Golodirsen, sold under the brand name Vyondys 53, is a medication used for the treatment of Duchenne muscular dystrophy.^[1]

70 relations: Abdominal pain, Abrasion (medicine), Accelerated approval (FDA), Adult, Back pain, Bruise, Chemistry, Child, Clearance (pharmacology), Clinical trial, Common cold, Comorbidity, Confirmatory trial, Constipation, Controversy, Cough, Diarrhea, Dizziness, Duchenne muscular dystrophy, Dystrophin, Ear, Eteplirsen, Exon, Exon skipping, Fast track (FDA), Fever, Food and Drug Administration, Glomerulonephritis, Headache, Indication (medicine), Infection, Influenza, Intravenous therapy, Kidney, Lactation, Ligament, Male, Male reproductive system, Medication, Monkey, Morpholino, Motor control, Mouse, Mutation, Nausea, Oligomer, Oligonucleotide, Orphan drug, Pain, Pathology, ... Expand index (20 more) »
Antisense RNA
Muscle protectors
Muscular dystrophy
Therapeutic gene modulation

Abdominal pain

Abdominal pain, also known as a stomach ache, is a symptom associated with both non-serious and serious medical issues.

See Golodirsen and Abdominal pain

Abrasion (medicine)

An abrasion is a partial thickness wound caused by damage to the skin.

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Accelerated approval (FDA)

The United States Food and Drug Administration (FDA) initiated the FDA Accelerated Approval Program in 1992 to allow faster approval of drugs for serious conditions that fill an unmet medical need.

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Adult

An adult is a human or other animal that has reached full growth.

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Back pain

Back pain (Latin: dorsalgia) is pain felt in the back.

See Golodirsen and Back pain

A bruise, also known as a contusion, is a type of hematoma of tissue, the most common cause being capillaries damaged by trauma, causing localized bleeding that extravasates into the surrounding interstitial tissues.

See Golodirsen and Bruise

Chemistry

Chemistry is the scientific study of the properties and behavior of matter.

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Child

A child is a human being between the stages of birth and puberty, or between the developmental period of infancy and puberty.

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Clearance (pharmacology)

In pharmacology, clearance (Cl_) is a pharmacokinetic parameter representing the efficiency of drug elimination.

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Clinical trial

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.

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Common cold

The common cold or the cold is a viral infectious disease of the upper respiratory tract that primarily affects the respiratory mucosa of the nose, throat, sinuses, and larynx.

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Comorbidity

In medicine, comorbidity refers to the simultaneous presence of two or more medical conditions in a patient; often co-occurring (that is, concomitant or concurrent) with a primary condition.

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Confirmatory trial

A confirmatory trial is an adequately controlled trial where hypotheses are stated in advance and evaluated according to a protocol.

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Constipation

Constipation is a bowel dysfunction that makes bowel movements infrequent or hard to pass.

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Controversy

Controversy is a state of prolonged public dispute or debate, usually concerning a matter of conflicting opinion or point of view.

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Cough

A cough is a sudden expulsion of air through the large breathing passages which can help clear them of fluids, irritants, foreign particles and microbes.

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Diarrhea

Diarrhea (American English), also spelled diarrhoea or diarrhœa (British English), is the condition of having at least three loose, liquid, or watery bowel movements in a day.

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Dizziness

Dizziness is an imprecise term that can refer to a sense of disorientation in space, vertigo, or lightheadedness.

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Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy predominantly affecting boys. Golodirsen and Duchenne muscular dystrophy are muscular dystrophy.

See Golodirsen and Duchenne muscular dystrophy

Dystrophin

Dystrophin is a rod-shaped cytoplasmic protein, and a vital part of a protein complex that connects the cytoskeleton of a muscle fiber to the surrounding extracellular matrix through the cell membrane. Golodirsen and Dystrophin are muscle protectors and muscular dystrophy.

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Ear

An ear is the organ that enables hearing and (in mammals) body balance using the vestibular system.

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Eteplirsen

Eteplirsen (brand name Exondys 51) is a medication to treat, but not cure, some types of Duchenne muscular dystrophy (DMD), caused by a specific mutation. Golodirsen and Eteplirsen are Antisense RNA, muscle protectors, muscular dystrophy and therapeutic gene modulation.

See Golodirsen and Eteplirsen

Exon

An exon is any part of a gene that will form a part of the final mature RNA produced by that gene after introns have been removed by RNA splicing.

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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.

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Fast track (FDA)

Fast track is a designation by the United States Food and Drug Administration (FDA) of an investigational drug for expedited review to facilitate development of drugs that treat a serious or life-threatening condition and fill an unmet medical need.

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Fever

Fever or pyrexia in humans is a body temperature above the normal range due to an increase in the body's temperature set point in the hypothalamus.

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Food and Drug Administration

The United States Food and Drug Administration (FDA or US FDA) is a federal agency of the Department of Health and Human Services.

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Glomerulonephritis

Glomerulonephritis (GN) is a term used to refer to several kidney diseases (usually affecting both kidneys).

See Golodirsen and Glomerulonephritis

Headache

Headache, also known as cephalalgia, is the symptom of pain in the face, head, or neck.

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Indication (medicine)

In medicine, an indication is a valid reason to use a certain test, medication, procedure, or surgery.

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Infection

An infection is the invasion of tissues by pathogens, their multiplication, and the reaction of host tissues to the infectious agent and the toxins they produce.

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Influenza

Influenza, commonly known as "the flu" or just "flu", is an infectious disease caused by influenza viruses.

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Intravenous therapy

Intravenous therapy (abbreviated as IV therapy) is a medical technique that administers fluids, medications and nutrients directly into a person's vein.

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Kidney

In humans, the kidneys are two reddish-brown bean-shaped blood-filtering organs that are a multilobar, multipapillary form of mammalian kidneys, usually without signs of external lobulation.

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Lactation

Lactation describes the secretion of milk from the mammary glands and the period of time that a mother lactates to feed her young.

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Ligament

A ligament is the fibrous connective tissue that connects bones to other bones.

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Male

Male (symbol: ♂) is the sex of an organism that produces the gamete (sex cell) known as sperm, which fuses with the larger female gamete, or ovum, in the process of fertilisation.

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Male reproductive system

The male reproductive system consists of a number of sex organs that play a role in the process of human reproduction.

See Golodirsen and Male reproductive system

Medication

A medication (also called medicament, medicine, pharmaceutical drug, medicinal drug or simply drug) is a drug used to diagnose, cure, treat, or prevent disease.

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Monkey

Monkey is a common name that may refer to most mammals of the infraorder Simiiformes, also known as the simians.

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Morpholino

A Morpholino, also known as a Morpholino oligomer and as a phosphorodiamidate Morpholino oligomer (PMO), is a type of oligomer molecule (colloquially, an oligo) used in molecular biology to modify gene expression.

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Motor control

Motor control is the regulation of movements in organisms that possess a nervous system.

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Mouse

A mouse (mice) is a small rodent.

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Mutation

In biology, a mutation is an alteration in the nucleic acid sequence of the genome of an organism, virus, or extrachromosomal DNA.

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Nausea

Nausea is a diffuse sensation of unease and discomfort, sometimes perceived as an urge to vomit.

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Oligomer

In chemistry and biochemistry, an oligomer is a molecule that consists of a few repeating units which could be derived, actually or conceptually, from smaller molecules, monomers.

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Oligonucleotide

Oligonucleotides are short DNA or RNA molecules, oligomers, that have a wide range of applications in genetic testing, research, and forensics.

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Orphan drug

An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. Golodirsen and orphan drug are orphan drugs.

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Pain

Pain is a distressing feeling often caused by intense or damaging stimuli.

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Pathology

Pathology is the study of disease and injury.

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Pharmacology

Pharmacology is the science of drugs and medications, including a substance's origin, composition, pharmacokinetics, pharmacodynamics, therapeutic use, and toxicology.

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Pharynx

The pharynx (pharynges) is the part of the throat behind the mouth and nasal cavity, and above the esophagus and trachea (the tubes going down to the stomach and the lungs respectively).

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Phosphoramidate

In organophosphorus chemistry, phosphoramidates (sometimes also called amidophosphates) are a class of phosphorus compounds structurally related to phosphates (or organophosphates) via the substitution of an group for an amine group.

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Placebo

A placebo is a substance or treatment which is designed to have no therapeutic value.

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Pregnancy

Pregnancy is the time during which one or more offspring develops (gestates) inside a woman's uterus (womb).

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Priority review

Priority review is a program of the United States Food and Drug Administration (FDA) to expedite the review process for drugs that are expected to have a particularly great impact on the treatment of a disease.

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Rhinitis

Rhinitis, also known as coryza, is irritation and inflammation of the mucous membrane inside the nose.

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Sarepta Therapeutics

Sarepta Therapeutics, Inc. is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States.

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Skin

Skin is the layer of usually soft, flexible outer tissue covering the body of a vertebrate animal, with three main functions: protection, regulation, and sensation.

See Golodirsen and Skin

Sprain

A sprain is a 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.

See Golodirsen and Sprain

Subcutaneous administration

Subcutaneous administration is the insertion of medications beneath the skin either by injection or infusion.

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Sue Fletcher

Sue Fletcher is a professor and lecturer at the University of Western Australia and a Senior Principal Research Fellow at Murdoch University.

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Surrogate endpoint

In clinical trials, a surrogate endpoint (or surrogate marker) is a measure of effect of a specific treatment that may correlate with a real clinical endpoint but does not necessarily have a guaranteed relationship.

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Tachycardia

Tachycardia, also called tachyarrhythmia, is a heart rate that exceeds the normal resting rate.

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Therapy

A therapy or medical treatment is the attempted remediation of a health problem, usually following a medical diagnosis.

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Toxicity

Toxicity is the degree to which a chemical substance or a particular mixture of substances can damage an organism.

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University of Western Australia

The University of Western Australia (UWA) is a public research university in the Australian state of Western Australia.

See Golodirsen and University of Western Australia

Vomiting

Vomiting (also known as emesis and throwing up) is the involuntary, forceful expulsion of the contents of one's stomach through the mouth and sometimes the nose.

See Golodirsen and Vomiting