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Vamorolone
Vamorolone (developmental code name VBP-15) is a synthetic steroid, which is under development for the treatment of Duchenne muscular dystrophy Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy that primarily affects boys. Muscle weakness usually begins around the age of four, and worsens quickly. Muscle loss typically occurs first in the thighs and pelvis follow .... Anti-inflammatory drugs of the corticosteroid class show a carbonyl (=O) or hydroxyl (-OH) group on the C11 carbon of the steroid backbone. In contrast, vamorolone contains a delta9,11 double bond between the C9 and C11 carbons. This change in structure has been shown to remove a molecular contact site with the glucocorticoid receptor, and lead to dissociative properties. Vamorolone is a partial agonist of the glucocorticoid receptor (''NR3C1'') with relative loss of transactivation activities, but retention of transrepression activities, compared to corticosteroidal drugs. In contrast ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a severe type of muscular dystrophy that primarily affects boys. Muscle weakness usually begins around the age of four, and worsens quickly. Muscle loss typically occurs first in the thighs and pelvis followed by the arms. This can result in trouble standing up. Most are unable to walk by the age of 12. Affected muscles may look larger due to increased fat content. Scoliosis is also common. Some may have intellectual disability. Females with a single copy of the defective gene may show mild symptoms. The disorder is X-linked recessive. About two thirds of cases are inherited from a person's mother, while one third of cases are due to a new mutation. It is caused by a mutation in the gene for the protein dystrophin. Dystrophin is important to maintain the muscle fiber's cell membrane. Genetic testing can often make the diagnosis at birth. Those affected also have a high level of creatine kinase in their blood. Although there is no know ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Synthetic Compound
In chemistry, organic compounds are generally any chemical compounds that contain carbon-hydrogen or carbon-carbon bonds. Due to carbon's ability to catenate (form chains with other carbon atoms), millions of organic compounds are known. The study of the properties, reactions, and syntheses of organic compounds comprise the discipline known as organic chemistry. For historical reasons, a few classes of carbon-containing compounds (e.g., carbonate salts and cyanide salts), along with a few other exceptions (e.g., carbon dioxide, hydrogen cyanide), are not classified as organic compounds and are considered inorganic. Other than those just named, little consensus exists among chemists on precisely which carbon-containing compounds are excluded, making any rigorous definition of an organic compound elusive. Although organic compounds make up only a small percentage of Earth's crust, they are of central importance because all known life is based on organic compounds. Living t ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Steroid
A steroid is a biologically active organic compound with four rings arranged in a specific molecular configuration. Steroids have two principal biological functions: as important components of cell membranes that alter membrane fluidity; and as signaling molecules. Hundreds of steroids are found in plants, animals and fungi. All steroids are manufactured in cells from the sterols lanosterol (opisthokonts) or cycloartenol (plants). Lanosterol and cycloartenol are derived from the cyclization of the triterpene squalene. The steroid core structure is typically composed of seventeen carbon atoms, bonded in four " fused" rings: three six-member cyclohexane rings (rings A, B and C in the first illustration) and one five-member cyclopentane ring (the D ring). Steroids vary by the functional groups attached to this four-ring core and by the oxidation state of the rings. Sterols are forms of steroids with a hydroxy group at position three and a skeleton derived from cholestane. ''A ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Diketones
In organic chemistry, a dicarbonyl is a molecule containing two carbonyl () groups. Although this term could refer to any organic compound containing two carbonyl groups, it is used more specifically to describe molecules in which both carbonyls are in close enough proximity that their reactivity is changed, such as 1,2-, 1,3-, and 1,4-dicarbonyls. Their properties often differ from those of monocarbonyls, and so they are usually considered functional groups of their own. These compounds can have symmetrical or unsymmetrical substituents on each carbonyl, and may also be functionally symmetrical (dialdehydes, diketones, diesters, ''etc.'') or unsymmetrical (keto-esters, keto-acids, ''etc.''). 1,2-Dicarbonyls 1,2-Dialdehyde The only 1,2-dialdehyde is glyoxal, . Like many alkyldialdehydes, glyoxal is encountered almost exclusively as its hydrate and oligomers thereof. These derivatives often behave equivalently to the aldehydes since hydration is reversible. Glyoxal condenses re ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Diols
A diol is a chemical compound containing two hydroxyl groups ( groups). An aliphatic diol is also called a glycol. This pairing of functional groups is pervasive, and many subcategories have been identified. The most common industrial diol is ethylene glycol. Examples of diols in which the hydroxyl functional groups are more widely separated include 1,4-butanediol and propylene-1,3-diol, or beta propylene glycol, . Synthesis of classes of diols Geminal diols A geminal diol has two hydroxyl groups bonded to the same atom. These species arise by hydration of the carbonyl compounds. The hydration is usually unfavorable, but a notable exception is formaldehyde which, in water, exists in equilibrium with methanediol H2C(OH)2. Another example is (F3C)2C(OH)2, the hydrated form of hexafluoroacetone. Many gem-diols undergo further condensation to give dimeric and oligomeric derivatives. This reaction applies to glyoxal and related aldehydes. Vicinal diols In a vicinal diol, t ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Glucocorticoids
Glucocorticoids (or, less commonly, glucocorticosteroids) are a class of corticosteroids, which are a class of steroid hormones. Glucocorticoids are corticosteroids that bind to the glucocorticoid receptor that is present in almost every vertebrate animal cell. The name "glucocorticoid" is a portmanteau (glucose + cortex + steroid) and is composed from its role in regulation of glucose metabolism, synthesis in the adrenal cortex, and its steroidal structure (see structure below). Glucocorticoids are part of the feedback mechanism in the immune system, which reduces certain aspects of immune function, such as inflammation. They are therefore used in medicine to treat diseases caused by an overactive immune system, such as allergies, asthma, autoimmune diseases, and sepsis. Glucocorticoids have many diverse (pleiotropic) effects, including potentially harmful side effects. They also interfere with some of the abnormal mechanisms in cancer cells, so they are used in high doses to tr ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
