The time it takes to absorb half of the chemical.
How long it takes the drug to get into circulation.
Access to Treatment Committee
The Committee at n-Lorem that reviews proposals from physicians to treat genetically confirmed nano-rare disease patients.
The function of adaptive immune responses is to destroy invading pathogens and any toxic molecules they produce.
adeno-associated virus (AAV Vector)
Part of the genetic information an associated virus has.
adenosine triphosphate (ATP)
A molecule that provides energy to cells and is involved in many cellular processes, including muscle contraction, protein synthesis, and cell division.
Produces adrenaline and noradrenaline and can make the plasma membrane more unstable, which results in an increase in heart rate.
The process by which cells convert oxygen and nutrients into energy (ATP), carbon dioxide, and water.
A suspension of fine solid or liquid particles in gas. In medicine, aerosols can be used to administer drugs.
A chemical that interacts with a receptor and causes a positive activation of the signaling system.
A hormone made in the adrenals that encourages the kidney to get rid of water.
One of two or more alternative forms of a gene that arise by mutation and are found at the same place on a chromosome.
Small, air-filled sacs in the lungs where gas exchange between the air and blood takes place.
The building blocks of proteins, consisting of organic compounds containing an amino group and a carboxyl group.
amyloid kidney disease
A type of kidney disease caused by the buildup of abnormal proteins (amyloid) in the kidneys, leading to kidney damage and dysfunction.
The process by which cells convert nutrients into energy without the use of oxygen.
A chemical substance that is the subject of chemical analysis.
A bulging, weakened area in the wall of a blood vessel that can rupture and cause serious bleeding or other complications.
A chemical that prevents the activation of a signaling system.
Chemicals that cause antibodies to be created.
antisense oligonucleotides (ASOs)
Short, synthetic, single-stranded oligodeoxynucleotides that can alter RNA and reduce, restore or modify protein expression through several distinct mechanisms.
The main artery in the body that carries oxygen-rich blood from the heart to the rest of the body.
A problem with the rate or rhythm of your heartbeat.
Small blood vessels that carry blood away from your heart; connectors between your arteries and capillaries.
A blood vessel that carries oxygen-rich blood away from the heart to the body's tissues.
A group of conditions characterized by inflammation and damage to the joints, causing pain, stiffness, and loss of mobility.
Collapse in alveoli that results in imbalances between air and blood flow.
Stiffening of arteries due to a buildup of fat or lipids on an arterial wall.
The more extreme version of atrial flutter.
A type of heart rhythm disorder (arrhythmia) caused by problems in the heart's electrical system.
A delay or disturbance in the transmission of an impulse from the atria to the ventricles.
Nerves that regulate involuntary body functions such as heart rate, digestion, and breathing.
autonomic nervous system
A component of the peripheral nervous system that regulates involuntary physiologic processes including heart rate, blood pressure, respiration, and digestion.
Drugs that are used to block adrenergic receptors when the heart is beating too fast.
The fraction of the dose administered that gets into the blood.
Chemicals that are produced by living organisms and are involved in various metabolic processes.
The manipulation of organisms or their components to produce useful products.
A bodily fluid that circulates through the heart, arteries, veins, and capillaries, carrying oxygen, nutrients, and waste products.
A slow heart rate.
The center of the nervous system, responsible for thought, emotion, and control of body functions.
An infection of the main airways of the lungs (bronchi), causing them to become irritated and inflamed. The main symptom is a cough, which may bring up yellow-grey mucus (phlegm).
Bronchospasms happen when the muscles that line your bronchi (airways in your lungs) tighten. This results in wheezing, coughing, and other symptoms.
A group of diseases characterized by the uncontrolled growth and spread of abnormal cells.
Small blood vessels that connect arteries and veins and allow for the exchange of oxygen, nutrients, and waste products between the blood and tissues.
The sudden stoppage of the heart.
The accumulation of fluid in the lungs causes difficulty breathing.
A closed circulatory system with a heart and branching network or arteries, capillaries, and veins.
A detailed report of the diagnosis, treatment, and follow-up of an individual patient. Case reports also contain some demographic information about the patient.
The smallest unit that can live on its own and that makes up all living organisms and the tissues of the body.
central nervous system (CNS)
The portion of the nervous system where signal integration occurs; In vertebrate animals, the brain and spinal cord.
A substance made up of elements, such as hydrogen or sodium.
Chemical networks refer to the complex systems of chemical reactions that occur within living organisms. These reactions involve the formation and breaking of chemical bonds and are essential for all biological processes.
Produces acetylcholine that reduces the rate on depolarization and repolarization, slowing the heart down.
A cellular structure consisting of one DNA molecule and associated protein molecules. (In some contexts, such as genome sequencing, the term may refer to the DNA alone).
chronic systemic inflammation
A low-grade, long-term immune response that is associated with many chronic diseases, such as heart disease, diabetes, and cancer. It can be caused by factors such as obesity, stress, and environmental toxins.
An establishment or hospital department where outpatients are given medical treatment or advice, especially of a specialist nature.
A three-nucleotide sequence of DNA or mRNA that specifies a particular amino acid or termination signal; the basic unit of the genetic code.
A protein found in the body's connective tissues, such as skin, tendons, ligaments, and bones. It provides strength and structure to these tissues and is essential for wound healing and tissue repair.
Part of your immune system that defends your body against injury and foreign invaders like bacteria and viruses that can make you sick. Your complement system activates proteins that work with your immune system to keep you healthy.
A measurement of how much pressure is required to increase lung volume.
The presence of two different mutated alleles at a particular gene locus.
Diseases that are present at birth.
congestive heart failure
A serious condition in which the heart doesn't pump blood as efficiently as it should.
Chemicals refer to any substance made up of one or more chemical compounds. Chemicals can be natural or synthetic, and are used in a wide range of industries, such as pharmaceuticals, agriculture, and manufacturing.
One course of treatment with a drug permanently reverses the patient from a disease phenotype to a different healthier phenotype.
The contents of the cell bounded by the plasma membrane.
A unit of mass used to express the molecular weight of proteins, nucleic acids, and other large molecules. One Dalton is defined as one-twelfth of the mass of a carbon-12 atom.
de novo mutations
Spontaneous mutations that occur in individuals. These mutations are not passed from parent to offspring.
A disease in which the structure or function of the effected tissues or organs changes for the worse over time.
deoxyribonucleic acid (DNA)
A nucleic acid molecule, usually a double-stranded helix, in which each polynucleotide strand consists of nucleotide monomers with a deoxyribose sugar and the nitrogenous base adenine (A), cytosine (C), guanine (G), and thymine (T); capable of being replicated and determining the inherited structure of a cell’s proteins.
The process of identifying a disease, condition, or injury from its signs and symptoms.
diastolic blood pressure
Blood pressure when your left ventricle is filling.
A disorder of structure or function in a human, animal, or plant.
In medicine, a disturbance of normal functioning of the mind or body.
distal convoluted tubule
A short nephron segment, interposed between the macula densa and collecting duct. Even though it is short, it plays a key role in regulating extracellular fluid volume and electrolyte homeostasis.
The amount of a medication or substance that is administered at one time or over a specified period. Dosing is typically determined based on factors such as the age, weight, and medical condition of the patient, as well as the intended therapeutic effect.
A graph representing the dose versus the dose-relationship.
Any substance (other than food) that is used to prevent, diagnose, treat, or relieve symptoms of a disease or abnormal condition. Drugs can also affect how the brain and rest of the body work and cause changes in mood, awareness, thoughts, feelings, or behavior.
duration of action
How long the drug lasts.
The half-maximal effective concentration.
The midpoint of a dose response curve.
The swelling caused by an accumulation of fluid in the body's tissues. Edema can be caused by a variety of factors, including heart failure, kidney disease, liver disease, and certain medications. Symptoms can include swelling, stiffness, and pain in the affected area.
The process by which the body gets rid of a drug.
The time it takes to get rid of half of a particular drug from an organ or from the whole body.
An enzyme which cleaves a polynucleotide chain by separating nucleotides other than the two end ones.
A thin layer of cells that lines the interior surface of blood vessels, as well as the heart and lymphatic vessels. It is responsible for regulating the passage of molecules and cells into and out of the bloodstream.
A measure of the amount of disorder or randomness in a system, such as a cell or organism. In biological systems, entropy tends to increase over time, leading to a breakdown of organization and function.
A segment of a DNA or RNA molecule containing information coding for a protein or peptide sequence.
Each of a pair of globular organs in the head through which people and vertebrate animals see, the visible part typically appearing almond-shaped in animals with eyelids.
Food and Drug Administration (FDA)
The administration in the United States that is responsible for protecting human health by ensuring the safety, efficacy, and security of human and veterinary drugs, biological products, and medical devices; and by ensuring the safety of the food supply, cosmetics, and products that emit radiation.
fraction of expired volume in one second (FEV1):
The amount of air expired in one second is called. FEV1 measures the ability to move air.
An abundant monosaccharide in the human diet that the body needs to metabolize.
The basic unit of heredity, composed of a specific sequence of DNA that codes for a particular protein or trait. Genes are responsible for determining many of an organism's characteristics, from eye color to susceptibility to certain diseases.
The introduction of genes into an afflicted individual for therapeutic purposes.
A genetic disease is a condition that is caused by an abnormality in an individual's DNA. This abnormality can be inherited from a parent, or it can occur spontaneously.
The scientific study of heredity and hereditary variation.
The genetic material of an organism or virus; the complete compliment of an organism’s or virus’s genes along with its noncoding nucleic acid sequences.
A laboratory method that is used to determine the entire genetic makeup of an organism or cell type.
The genetic makeup, or set of alleles, of an organism.
glomerular filtration rate (GFR)
The rate which blood is filtered.
Tiny structures in the kidneys that filter blood and remove waste products from the body. They are made up of small blood vessels called capillaries and play an important role in maintaining normal kidney function.
A condition in which the glomeruli in the kidneys become damaged or scarred, leading to impaired kidney function. This can be caused by a number of factors, including high blood pressure, diabetes, and certain autoimmune diseases.
A hormone that is produced by the pancreas and plays an important role in regulating blood sugar levels in the body. It stimulates the liver to release glucose into the bloodstream, which can help to counteract low blood sugar levels.
A type of sugar that is found in many foods and is the primary source of energy for the body's cells. It is transported through the bloodstream and is used by cells to produce ATP, which is the energy currency of the body.
A metabolic pathway that occurs in the cytoplasm of cells and is responsible for breaking down glucose into smaller molecules that can be used to produce ATP. It is the first step in cellular respiration and is an essential process for the production of energy in the body.
Goblet cells arise from pluripotent stem cells and derive their name from their goblet, cup-like appearance. The primary function of goblet cells is to secrete mucin and create a protective mucus layer.
Good Manufacturing Practices (GMP)
Good manufacturing practices enforced by the FDA.
guanosine triphosphate (GTP)
A molecule used by cells to store energy. It is similar to ATP but plays a different role in the cell.
The organized provision of medical care to individuals or a community.
A muscular organ that pumps blood throughout the body. It is responsible for providing oxygen and nutrients to the body's tissues and removing waste products.
Occurs when the flow of blood to the heart is severely reduced or blocked. The blockage is usually due to a buildup of fat, cholesterol and other substances in the heart (coronary) arteries.
The protein contained in red blood cells that is responsible for delivery of oxygen to the tissues.
Medical doctors who diagnose, treat, and manage problems associated with your liver, gallbladder, bile ducts, and pancreas.
Having two different alleles of a particular gene or genes.
A state of balance among all the body systems needed to survive and function correctly. In homeostasis, body levels of acid, blood pressure, blood sugar, electrolytes, energy, hormones, oxygen, proteins, and temperature are constantly adjusted to respond to changes inside and outside the body, to keep them at a normal level.
Having two identical alleles of a particular gene or genes.
A chemical substance produced by the body that regulates various bodily functions, such as growth, metabolism, and reproduction. Hormones are typically released by glands, such as the thyroid or adrenal glands, and carried through the bloodstream to their target cells.
Hydrophilic substances are attracted to water and are able to dissolve in it. These substances typically have polar or charged molecules that allow them to interact with water molecules.
Hydrophobic substances are repelled by water and are unable to dissolve in it. These substances typically have nonpolar molecules that do not interact well with water molecules.
The increased thickness of the stratum corneum, the outer layer of the skin.
hypertension (high blood pressure)
High blood pressure develops when blood flows through your arteries at higher-than-normal pressures.
The half-maximal inhibitory concentration.
Medications that suppress the immune system to prevent it from attacking transplanted organs, treating autoimmune disorders, or preventing rejection of transplanted tissue. These medications can also increase the risk of infection and certain types of cancer.
inferior vena cava
The largest vein in your body.
A natural process in the body that is characterized by redness, swelling, warmth, pain, and sometimes loss of function. It is the body's response to injury, infection, or irritation, and involves a complex network of cells and chemicals that work together to protect the body from harmful stimuli.
Innate, or nonspecific, immunity is the defense system with which you were born. It protects you against all antigens. Innate immunity involves barriers that keep harmful materials from entering your body.
institutional review board (IRB)
A group that has been formally designated to review and monitor biomedical research involving human subjects.
A hormone produced by the pancreas that regulates the level of glucose in the blood. Insulin helps glucose to enter the body's cells, where it is used for energy or stored for future use. In people with diabetes, the body either does not produce enough insulin or does not respond to it properly, leading to high blood sugar levels.
The outer covering of a mammal’s body, including skin, hair, and nails, claws, or hooves.
Situated or taking place within, or administered into, a muscle.
The fluid filled space between the thin layers of tissue that cover the brain and spinal cord.
Existing or taking place within, or administered into, a vein or veins.
A shot of medicine to the eye.
investigational new drug (IND)
A substance that has been tested in the laboratory and has been approved by the U.S. Food and Drug Administration (FDA) for testing in people. Clinical trials test how well investigational new drugs work and whether they are safe to use. An investigational new drug may be approved by the FDA for use in one disease or condition but still be considered investigational in other diseases or conditions. Also called experimental drug, IND, investigational agent, and investigational drug.
An atom or molecule that has an electrical charge, either positive or negative, due to the gain or loss of one or more electrons. Ions play a critical role in many physiological processes, such as nerve and muscle function, and the transport of nutrients and waste products across cell membranes.
The specialized cells in the kidney that are located near the glomerulus and are responsible for regulating blood pressure and kidney function. These cells produce and release a hormone called renin, which helps to control blood pressure by activating the renin-angiotensin-aldosterone system.
One of a pair of organs in the abdomen. The kidneys remove waste and extra water from the blood (as urine) and help keep chemicals (such as sodium, potassium, and calcium) balanced in the body. The kidneys also make hormones that help control blood pressure and stimulate bone marrow to make red blood cells.
Small, hard deposits that form in the kidneys from the buildup of minerals and salts in the urine. Kidney stones can cause severe pain, nausea, and vomiting, and can sometimes lead to infection or damage to the kidneys. Treatment options may include pain management, increased fluid intake, or surgical removal of the stones.
The Krebs cycle, also known as the citric acid cycle or tricarboxylic acid (TCA) cycle, is a series of chemical reactions that occur in the mitochondria of cells and are involved in the production of ATP, the body's main source of energy. The Krebs cycle begins with the breakdown of glucose and ends with the production of carbon dioxide and water, as well as ATP.
A substance made by muscle tissue and by red blood cells, which carry oxygen from your lungs to other parts of your body. Normally, the level of lactic acid in the blood is low. Lactic acid levels rise when oxygen levels decrease.
left anterior descending artery (LAD)
An artery which supplies blood to the left ventricle.
Lipids are a group of naturally occurring molecules that include fats, waxes, sterols, and fat-soluble vitamins. They are a major component of cell membranes and play a crucial role in energy storage and hormone production.
A large organ located in the upper abdomen. The liver cleanses the blood and aids in digestion by secreting bile.
loop of Henle
A section of the nephron in the kidney that plays a key role in the production of urine. It is responsible for reabsorbing water and sodium from the filtrate and creating a concentration gradient in the surrounding tissues.
One of a pair of organs in the chest that supplies the body with oxygen, and removes carbon dioxide from the body.
A lymph is a clear fluid that circulates through the lymphatic system, which is part of the immune system. It contains white blood cells, nutrients, and waste products, and helps to remove toxins and foreign substances from the body.
Freeze dry (a substance).
The maximum effect a drug can produce.
Refers to the practices and procedures used for the prevention, treatment, or relief of symptoms of diseases or abnormal conditions. This term may also refer to a legal drug used for the same purpose.
The connection between the brainstem and the spinal cord, carrying multiple important functional centers. It is comprised of the cardiovascular-respiratory regulation system, descending motor tracts, ascending sensory tracts, and origin of cranial nerves IX, X, XI, and XII.
messenger RNA (mRNA)
A type of RNA found in cells. mRNA molecules carry the genetic information needed to make proteins. They carry the information from the DNA in the nucleus of the cell to the cytoplasm where the proteins are made.
The chemical changes that take place in a cell or an organism. These changes make energy and the materials cells and organisms need to grow, reproduce, and stay healthy. Metabolism also helps get rid of toxic substances.
A type of RNA found in cells and in blood. MicroRNAs are smaller than many other types of RNA and can bind to messenger RNAs (mRNAs) to block them from making proteins.
Membrane-enclosed organelles in the cytoplasm of most eukaryotic cells. The site of cellular energy production (ATP).
Two or more atoms that are held together by covalent bonds
monoclonal antibody (mAb)
A type of medication that is made by identical immune cells that are all clones of a unique parent cell. These medications are designed to target specific antigens on cancer cells or other abnormal cells in the body.
A molecule that can be bonded to other identical molecules to form a polymer.
Long, slender cells that make up muscle tissue and are capable of contracting and relaxing to produce movement.
A change in the nucleotide sequence of an organism’s DNA or in the DNA or RNA of a virus.
A disease caused by a genetic mutation that affects less than 30 people in the world.
the branch of medicine that deals with the physiology and diseases of the kidneys.
The branch of medicine that deals with the diagnosis and treatment of disorders of the nervous system.
A molecule that forms part of the building blocks of DNA and RNA, and includes adenine, guanine, cytosine, thymine, and uracil.
A complex molecule that carries genetic information in the form of DNA or RNA, and plays a key role in protein synthesis and cellular function.
Nucleotides are the building blocks of nucleic acids like DNA and RNA. They consist of three components: a nitrogenous base, a pentose sugar, and a phosphate group. The nitrogenous base can be adenine, guanine, cytosine, or thymine in DNA, or uracil in RNA.
(1) An atoms central core, containing protons and neutrons. (2) The organelle of a eukaryotic cell that contains the genetic material in the form of chromosomes, made up of chromatin. (3) A cluster of neurons.
A mutation that results in either no gene product or the absence of function at the phenotypic level.
An oligomer is a molecule made up of a small number of repeating units, called monomers. In biochemistry, oligomers usually consist of fewer than 10 monomers, while polymers consist of many more.
An oligonucleotide is a short DNA or RNA molecule consisting of a sequence of nucleotides, typically fewer than 50. Oligonucleotides are used in many areas of biological research, including gene expression analysis, genetic testing, and as therapeutic agents.
onset of action
How fast a drug produces its effect.
The branch of medicine concerned with the diagnosis and treatment of disorders of the eye.
An organ is a group of tissues that work together to perform a specific function in the body. Organs can be found in both plants and animals and perform a wide range of functions, such as digestion, respiration, and circulation.
the amount of solute in a solvent
A cellular process that harnesses the reduction of oxygen to generate high-energy phosphate bonds in the form of adenosine triphosphate (ATP).
parasympathetic nervous system
Your parasympathetic nervous system is a network of nerves that relaxes your body after periods of stress or danger. It also helps run life-sustaining processes, like digestion, during times when you feel safe and relaxed.
paroxysmal ventricular contractions
These contractions when the ventricle beats on its own. Frequent paroxysmal ventricular contractions can disrupt normal electrical activity and can lead to ventricular fibrillation.
particulate matter (particulates)
Particles (tiny pieces) of solids or liquids that are in the air.
A genetic alteration that increases an individual’s susceptibility or predisposition to a certain disease or disorder. When such a variant (or mutation) is inherited, development of symptoms is more likely, but not certain.
The disordered physiological processes associated with disease or injury.
A person receiving or registered to receive medicinal treatment.
Swelling and irritation of the thin, saclike tissue surrounding the heart (pericardium).
The lining of the heart.
peripheral circulatory system
The part of the circulatory system that carries blood from the heart to the arms, legs, and other body parts. It includes the arteries, which carry oxygen-rich blood away from the heart, and the arterioles and capillaries, which deliver oxygen and nutrients to the body's tissues.
peripheral nervous system (PMS)
The sensory and motor neurons that connect to the central nervous system.
The coordinated, rhythmic muscle contractions that move food through the digestive tract. Peristalsis occurs throughout the digestive tract, from the esophagus to the rectum, and is essential for proper digestion and elimination of waste.
pH (potential hydrogen)
A measure of the acidity or alkalinity of a solution, with a range of 0 to 14. A pH of 7 is considered neutral, while pH values below 7 are acidic and pH values above 7 are alkaline. pH is an important factor in many biological processes, and deviations from normal pH can have adverse effects on the body.
A compound manufactured for use as a medicinal drug.
The effects of a drug on the body, including how the drug interacts with its target receptor or enzyme, as well as any other physiological changes that result from the drug's activity. Pharmacodynamics is one of the two main branches of pharmacology, the other being pharmacokinetics
The way a drug is absorbed, distributed, metabolized, and eliminated by the body. Pharmacokinetics is one of the two main branches of pharmacology, the other being pharmacodynamics. Understanding a drug's pharmacokinetic properties is important for determining the appropriate dosing regimen and avoiding adverse effects.
The physical and observable characteristics of an organism, including its morphology, behavior, and other traits. Phenotype is determined by both genetic and environmental factors, and can vary widely within and between populations. Phenotype is an important concept in genetics, evolutionary biology, and many other fields of biology.
Phosphate groups are chemical groups consisting of a phosphorus atom bonded to four oxygen atoms. They play a crucial role in many biochemical processes, including energy transfer, DNA and RNA synthesis, and signal transduction.
A person qualified to practice medicine.
A pair of membranes that surrounds the lungs.
An infection that inflames the air sacs in one or both lungs. The air sacs may fill with fluid or pus (purulent material), causing cough with phlegm or pus, fever, chills, and difficulty breathing. A variety of organisms, including bacteria, viruses and fungi, can cause pneumonia.
polyclonal antibody (pAb)
Polyclonal antibodies are antibodies that are produced by different B cells in response to an antigen, resulting in a mixture of antibodies that can recognize and bind to different epitopes (parts) of the antigen. They are used in research and diagnostics, as well as in the treatment of certain diseases.
A polymer is a large molecule made up of repeating subunits (monomers) that are linked together by covalent bonds. Polymers can be natural or synthetic, and they have a wide range of applications in materials science, medicine, and industry.
Proteins are complex biomolecules that perform a wide range of functions in the body, including catalyzing chemical reactions, transporting molecules, providing structure to cells and tissues, and facilitating communication between cells. They are made up of long chains of amino acids that are folded into specific three-dimensional structures.
protein wasting nephropathy
A condition characterized by the loss of protein in the urine due to damage to the kidneys. It can be caused by various underlying conditions, including diabetes, high blood pressure, and autoimmune disorders, and it can lead to malnutrition and other complications if left untreated.
proximal convoluted tubule
A part of the nephron, the functional unit of the kidney. It is located in the renal cortex and is responsible for reabsorbing water, ions, and nutrients from the filtrate that is produced by the glomerulus.
Arteries which carry blood from your heart to your lungs.
A blockage of one or more arteries in the lungs, usually caused by a blood clot that has traveled from another part of the body. It can be life-threatening and requires prompt medical attention. Symptoms can include chest pain, shortness of breath, and coughing up blood.
The accumulation of gunk on alveolar membranes, or stiffening of the lung, or anything that interferes with the movement of the diaphragm and ancillary respiratory muscles, and can alter pulmonary function.
A type of high blood pressure that affects the arteries in the lungs and the right side of the heart.
The system of organs and tissues that are involved in breathing. It includes the nose, mouth, trachea, bronchi, and lungs, as well as the muscles and nerves that control breathing. Its primary function is to exchange oxygen and carbon dioxide between the body and the environment.
A branch of medicine that specializes in diagnosing and treating diseases of the lungs and other parts of the respiratory system. These diseases include asthma, emphysema, tuberculosis, and pneumonia.
Consists of the electrical system of the heart. It controls how fast the heart beats.
A bacterial infection of the kidneys, usually caused by a urinary tract infection that spreads to the upper urinary tract. It can cause fever, back pain, and other symptoms and can lead to serious complications if left untreated.
A molecule that is produced during the breakdown of glucose in the body. It plays a key role in energy metabolism and is an important intermediate in many biochemical pathways.
A receptor is a protein molecule on the surface of a cell that can bind to a specific molecule, such as a hormone, neurotransmitter, or drug, and transmit a signal to the inside of the cell. Receptors play a crucial role in many physiological processes, including sensory perception, hormone signaling, and immune response.
The amount of air left in the lungs after expiration.
ribonucleic acid (RNA)
Ribonucleic acid is a type of nucleic acid that is involved in the synthesis of proteins in the body. It is transcribed from DNA and carries genetic information from the nucleus to the ribosomes, where it is translated into protein. RNA also plays other important roles in gene expression and regulation.
The structure containing RNA and its bound proteins.
A complex of rRNA and protein molecules that functions as a site of protein synthesis in the cytoplasm; consists of a large and a small subunit.
RNA interference (RNAi)
A mechanism for silencing the expression of specific genes. In RNAi, double-stranded RNA molecules that match the sequence of a particular gene are processed into siRNAs that either block translation or trigger the degradation of the gene’s messenger RNA. This happens naturally in some cells, and can be carried out in laboratory experiments as well.
RNA targeted drug
An RNA targeted drug is a medication that is designed to specifically target and modulate the function of RNA molecules, such as messenger RNA (mRNA) or microRNA (miRNA), in order to treat or prevent disease. RNA targeted drugs are a promising area of research for a wide range of conditions, including cancer, genetic disorders, and viral infections.
SGLT II (sodium glucose cotransporter receptor II)
A protein that is involved in the reabsorption of glucose from the urine in the kidneys. It is a target for some medications used to treat type 2 diabetes, known as SGLT2 inhibitors.
A class of medications used to treat type 2 diabetes by blocking the action of the SGLT2 protein in the kidneys, which leads to increased glucose excretion in the urine and lower blood glucose levels. They can also have other beneficial effects on cardiovascular and renal health.
single-nucleotide polymorphisms (SNPs)
A DNA sequence variation that occurs when a single nucleotide in the genome sequence is altered and the particular alteration is present in at least 1% of the population.
sinoatrial (SA) node
An area of cells in the atrium that undergo rapid depolarization and repolarization cycles.
The framework that supports the soft tissues of vertebrate animals and protects many of their internal organs. The skeletons of vertebrates are made of bone and/or cartilage.
small interfering RNA (siRNA)
One of multiple small, single-stranded RNA molecules generated by cellular machinery from a long, linear, double-stranded RNA molecule. The siRNA associates with one or more proteins in a complex that can degrade or prevent translation of an mRNA with a complimentary sequence.
small molecule drug (SMD)
A small molecule drug is a medication that consists of a relatively small organic molecule, typically less than 500-1000 Daltons in size, and is able to bind to specific target molecules in the body, such as enzymes, receptors, or transporters. Small molecule drugs are a common type of medication and are used to treat a wide range of conditions.
A medical emergency that occurs when blood flow to the brain is interrupted, either by a blood clot or a ruptured blood vessel. This can cause brain damage and a range of symptoms, including weakness or paralysis, difficulty speaking or understanding speech, and loss of vision. Prompt treatment is essential to minimize the damage caused by a stroke.
Study Treatment and Assessment Review Committee (STAR)
The committee at n-Lorem that provides n-Lorem and the patient’s physicians guidance on the development of treatment goals and clinical outcome assessments for n-Lorem’s diverse patient population.
Beneath the skin.
A disaccharide, or two-part molecule, formed by linking the monosaccharide sugars glucose and fructose.
A type of carbohydrate that is found in many foods, including fruits, vegetables, and grains, as well as in processed foods and sweetened beverages.
sympathetic nervous system
The sympathetic nervous system is best known for its role in responding to dangerous or stressful situations. In these situations, your sympathetic nervous system activates to speed up your heart rate, deliver more blood to areas of your body that need more oxygen or other responses to help you get out of danger.
A physical or mental problem that a person experiences that may indicate a disease or condition.
A type of white blood cell called lymphocytes. They help your immune system fight germs and protect you from disease.
The midpoint of a dose response curve for a toxic effect.
tertiary care center
A hospital that provides highly specialized medical services, such as neurosurgery, cardiac surgery, cancer treatment, and organ transplantation, to patients referred from other hospitals or primary care physicians. These centers have advanced medical equipment, highly trained medical professionals, and research facilities to diagnose and treat complex medical conditions.
The purpose for which we administer a drug.
therapeutic index (TI)
The therapeutic index is the range of doses at which a medication is effective without unacceptable adverse events. Drugs with a narrow TI (NTIDs) have a narrow window between their effective doses and those at which they produce adverse toxic effects.
A integrated group of cells with a common structure, function, or both.
The study of the effects of chemical, physical, or biological agents on living organisms and the environment. Toxicologists evaluate the toxicity of substances and determine safe levels of exposure to protect human health and the environment. They use various methods to identify, measure, and assess the risks associated with toxic substances, including animal studies, epidemiological studies, and computer modeling.
The process by which the genetic information stored in DNA is copied into RNA. This process involves the synthesis of messenger RNA (mRNA) by RNA polymerase, using one strand of DNA as a template. Transcription is a critical step in gene expression, as the mRNA transcript carries the genetic information to the ribosome, where it is translated into a protein.
The synthesis of a polypeptide using the genetic information encoded in an mRNA molecule. There is a change of “language” from nucleotides to amino acids.
type one diabetes
A chronic autoimmune disorder in which the body's immune system attacks and destroys the insulin-producing beta cells in the pancreas. This results in a lack of insulin, a hormone that regulates blood sugar levels. Type one diabetes typically develops in childhood or adolescence, although it can occur at any age. Treatment involves insulin replacement therapy, blood sugar monitoring, and lifestyle modifications to prevent complications.
type two diabetes
A chronic metabolic disorder in which the body becomes resistant to insulin or does not produce enough insulin to maintain normal blood sugar levels. It is usually diagnosed in adults, although it can occur at any age. Risk factors for type two diabetes include obesity, physical inactivity, and a family history of the disease. Treatment involves lifestyle modifications, such as weight loss and regular exercise, as well as medications to lower blood sugar levels.
Undiagnosed Disease Network (UDN)
The Undiagnosed Diseases Network (UDN) is a research study that is funded by the National Institutes of Health Common Fund. Its purpose is to bring together clinical and research experts from across the United States to solve challenging medical mysteries using advanced technologies.
A waste product that is formed when the body breaks down purines, which are found in many foods and are also produced by the body. Normally, uric acid is dissolved in the blood and excreted in the urine. However, if the body produces too much uric acid or if the kidneys cannot excrete it properly, uric acid can accumulate in the blood, leading to a condition known as hyperuricemia. This can cause gout, a type of arthritis, and kidney stones.
A peptide hormone made in the brain that encourages the kidney to resorb more water.
A blood vessel that carries blood back to the heart. Veins have thin walls and contain valves that help prevent the backflow of blood. There are three types of veins in the body: superficial veins, which are located just beneath the skin; deep veins, which are located within the muscles; and perforating veins, which connect the superficial and deep veins.
A type of arrhythmia, or irregular heartbeat, that affects your heart's ventricles. Ventricular fibrillation is life-threatening and requires immediate medical attention.
A very simple microorganism that infects cells and may cause disease. Because viruses can multiply only inside infected cells, they are not considered to be alive.
whole genome sequencing (WGS)
Whole genome sequencing is a laboratory procedure that determines the order of nucleotide bases (A, T, C, and G) in the genome of an organism in one process.