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Sobetirome
Sobetirome (GC-1) is a thyromimetic drug that binds to the thyroid hormone receptor TRβ1 preferentially compared to TRα1. It has been investigated for the treatment of dyslipidemia, obesity, Pitt–Hopkins syndrome, cholestatic liver disease, multiple sclerosis, bleomycin-induced lung fibrosis, and COVID-19 caused ARDS. It was designated as an orphan drug by the FDA for the treatment of X-linked adrenoleukodystrophy Adrenoleukodystrophy (ALD) is a disease linked to the X chromosome. It is a result of fatty acid buildup caused by failure of peroxisomal fatty acid beta oxidation which results in the accumulation of very long chain fatty acids in tissues throu .... References {{reflist Orphan drugs Thyroid hormone receptor beta agonists Carboxylic acids Phenols Isopropyl compounds ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Thyromimetic
Thyromimetic drugs are synthetic agonists of the thyroid hormone receptor's isoforms TR α1, TRα2, TRβ1, or TRβ2, mimicking some or all of the effects of endogenously produced thyroid hormones that regulate metabolism. Some thyromimetic drugs are selective for various of these receptors over others, enabling more targeted effects and reducing toxicity. Thyromimetics selective for TRβ—including eprotirome, sobetirome, resmetirom, and the prodrug VK2809—have been investigated for the treatment of non-alcoholic fatty liver disease, dyslipidemia Dyslipidemia is a metabolic disorder characterized by abnormally high or low amounts of any or all lipids (e.g. fats, triglycerides, cholesterol, phospholipids) or lipoproteins in the blood. Dyslipidemia is a risk factor for the development of ..., and other metabolic and neurodegenerative diseases. References Further reading *{{cite journal , last1=Yehuda-Shnaidman , first1=Einav , last2=Kalderon , first2=Bella , last3=Bar-Tan ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Oral Administration
Oral administration is a route of administration whereby a substance is taken through the Human mouth, mouth, swallowed, and then processed via the digestive system. This is a common route of administration for many medications. Oral administration can be easier and less painful than other routes of administration, such as Injection (medicine), injection. However, the onset of action is relatively low, and the effectiveness is reduced if it is not absorbed properly in the digestive system, or if it is broken down by digestive enzymes before it can reach the bloodstream. Some medications may cause gastrointestinal side effects, such as nausea or vomiting, when taken orally. Oral administration can also only be applied to conscious patients, and patients able to swallow. Terminology ''Per os'' (; ''P.O.'') is an adverbial phrase meaning literally from Latin "through the mouth" or "by mouth". The expression is used in medicine to describe a treatment that is taken orally (but not ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Carboxylic Acids
In organic chemistry, a carboxylic acid is an organic acid that contains a carboxyl group () attached to an Substituent, R-group. The general formula of a carboxylic acid is often written as or , sometimes as with R referring to an organyl group (e.g., alkyl, alkenyl, aryl), or hydrogen, or other groups. Carboxylic acids occur widely. Important examples include the amino acids and fatty acids. Deprotonation of a carboxylic acid gives a carboxylate anion. Examples and nomenclature Carboxylic acids are commonly identified by their trivial names. They often have the suffix ''-ic acid''. IUPAC-recommended names also exist; in this system, carboxylic acids have an ''-oic acid'' suffix. For example, butyric acid () is butanoic acid by IUPAC guidelines. For nomenclature of complex molecules containing a carboxylic acid, the carboxyl can be considered position one of the parent chain even if there are other substituents, such as 3-chloropropanoic acid. Alternately, it can be named ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Thyroid Hormone Receptor Beta Agonists
The thyroid, or thyroid gland, is an endocrine gland in vertebrates. In humans, it is a butterfly-shaped gland located in the neck below the Adam's apple. It consists of two connected lobes. The lower two thirds of the lobes are connected by a thin band of tissue called the isthmus (: isthmi). Microscopically, the functional unit of the thyroid gland is the spherical thyroid follicle, lined with follicular cells (thyrocytes), and occasional parafollicular cells that surround a lumen containing colloid. The thyroid gland secretes three hormones: the two thyroid hormones triiodothyronine (T3) and thyroxine (T4)and a peptide hormone, calcitonin. The thyroid hormones influence the metabolic rate and protein synthesis and growth and development in children. Calcitonin plays a role in calcium homeostasis. Secretion of the two thyroid hormones is regulated by thyroid-stimulating hormone (TSH), which is secreted from the anterior pituitary gland. TSH is regulated by thyrotro ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drugs
An orphan drug is a pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation (if there is any) of the country. Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development. Examples of this can be that in the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
X-linked Adrenoleukodystrophy
Adrenoleukodystrophy (ALD) is a disease linked to the X chromosome. It is a result of fatty acid buildup caused by failure of peroxisomal fatty acid beta oxidation which results in the accumulation of very long chain fatty acids in tissues throughout the body. The most severely affected tissues are the myelin in the central nervous system, the adrenal cortex, and the Leydig cells in the testes. The long chain fatty acid buildup causes damage to the myelin sheath of the neurons of the brain, resulting in seizures and hyperactivity. Other symptoms include problems in speaking, listening, and understanding verbal instructions. Clinically, ALD presents as a heterogeneous disorder, showing several distinct phenotypes, and no clear pattern of genotype–phenotype correlation. As an X-linked disorder, ALD presents more frequently and severely in males; however, approximately 80% of heterozygote females show some symptoms later in life. Approximately one third of male ALD patients wi ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Orphan Drug
An orphan drug is a medication, pharmaceutical agent that is developed to treat certain rare medical conditions. An orphan drug would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions that orphan drugs are used to treat are referred to as orphan diseases. The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the legislation (if there is any) of the country. Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug medical research, research and development. Examples of this can be that in the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to en ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
ARDS
Ards (or ARDS, ARDs) may refer to: Medical * ARDS, Acute respiratory distress syndrome * ARDs, age-related diseases Places * Ards Peninsula, Northern Ireland ** Ards (territory), several historical territorial divisions on the Ards Peninsula *** Ards Lower, a barony in Northern Ireland *** Ards Upper, a barony in Northern Ireland ** Ards and North Down, current administrative district on the Ards Peninsula *** Ards (borough), former administrative district *** Ards (Northern Ireland Parliament constituency), former constituency ** Ards Forest Park, a nature reserve in County Donegal, Ireland ** Newtownards Newtownards (; ) is a town in County Down, Northern Ireland. It lies at the most northern tip of Strangford Lough, 10 miles (16 km) east of Belfast, on the Ards Peninsula. It is in the Civil parishes in Ireland, civil parish of Newtow ..., a town in Northern Ireland, nicknamed "Ards" Organizations * Ards Community Hospital, a health facility in Newtownar ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Lung Fibrosis
Pulmonary fibrosis is a condition in which the lungs become scarred over time. Symptoms include shortness of breath, a dry cough, feeling tired, weight loss, and nail clubbing. Complications may include pulmonary hypertension, respiratory failure, pneumothorax, and lung cancer. Causes include environmental pollution, certain medications, connective tissue diseases, infections, and interstitial lung diseases. But in most cases the cause is Idiopathic disease, unknown (idiopathic pulmonary fibrosis). Diagnosis may be based on symptoms, medical imaging, lung biopsy, and lung function tests. No cure exists and treatment options are limited. Treatment is directed toward improving symptoms and may include oxygen therapy and pulmonary rehabilitation. Certain medications may slow the scarring. Lung transplantation may be an option. At least 5 million people are affected globally. Life expectancy is generally less than five years. Signs and symptoms Symptoms of pulmonary fibrosis ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
COVID-19
Coronavirus disease 2019 (COVID-19) is a contagious disease caused by the coronavirus SARS-CoV-2. In January 2020, the disease spread worldwide, resulting in the COVID-19 pandemic. The symptoms of COVID‑19 can vary but often include fever, fatigue, cough, breathing difficulties, anosmia, loss of smell, and ageusia, loss of taste. Symptoms may begin one to fourteen days incubation period, after exposure to the virus. At least a third of people who are infected asymptomatic, do not develop noticeable symptoms. Of those who develop symptoms noticeable enough to be classified as patients, most (81%) develop mild to moderate symptoms (up to mild pneumonia), while 14% develop severe symptoms (dyspnea, hypoxia (medical), hypoxia, or more than 50% lung involvement on imaging), and 5% develop critical symptoms (respiratory failure, shock (circulatory), shock, or organ dysfunction, multiorgan dysfunction). Older people have a higher risk of developing severe symptoms. Some complicati ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Bleomycin
-13- (1''H''-imidazol-5-yl)methyl9-hydroxy-5- 1''R'')-1-hydroxyethyl8,10-dimethyl-4,7,12,15-tetraoxo-3,6,11,14-tetraazapentadec-1-yl}-2,4'-bi-1,3-thiazol-4-yl)carbonyl]amino}propyl)(dimethyl)sulfonium , C=55 , H=84 , N=17 , O=21 , S=3 , SMILES = CC1=C(N=C(N=C1N) @HCC(=O)N)NC @@HC(=O)N)N)C(=O)N @@HC(C2=CN=CN2)O @H @H @H @@H @@HO3)CO)O)O)O @@H @H @H @@H @HO4)CO)O)OC(=O)N)O)C(=O)N @HC) @H @HC)C(=O)N @@H @@HC)O)C(=O)NCCC5=NC(=CS5)C6=NC(=CS6)C(=O)NCCC +C)C)O , StdInChI_Ref = , StdInChI = 1S/C55H83N17O21S3/c1-20-33(69-46(72-44(20)58)25(12-31(57)76)64-13-24(56)45(59)82)50(86)71-35(41(26-14-61-19-65-26)91-54-43(39(80)37(78)29(15-73)90-54)92-53-40(81)42(93-55(60)88)38(79)30(16-74)89-53)51(87)66-22(3)36(77)21(2)47(83)70-34(23(4)75)49(85)63-10-8-32-67-28(18-94-32)52-68-27(17-95-52)48(84)62-9-7-11-96(5)6/h14,17-19,21-25,29-30,34-43,53-54,64,73-75,77-81H,7-13,15-16,56H2,1-6H3,(H13-,57,58,59,60,61,62,63,65,66,69,70,71,72,76,82,83,84,85,86,87,88)/p+1/t21-,22+,23+,24-, ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
Multiple Sclerosis
Multiple sclerosis (MS) is an autoimmune disease resulting in damage to myelinthe insulating covers of nerve cellsin the brain and spinal cord. As a demyelinating disease, MS disrupts the nervous system's ability to Action potential, transmit signals, resulting in a range of signs and symptoms, including physical, cognitive disability, mental, and sometimes psychiatric problems. Symptoms include double vision, vision loss, eye pain, muscle weakness, and loss of Sensation (psychology), sensation or coordination. MS takes several forms, with new symptoms either occurring in isolated attacks (relapsing forms) or building up over time (progressive forms). In relapsing forms of MS, symptoms may disappear completely between attacks, although some permanent neurological problems often remain, especially as the disease advances. In progressive forms of MS, bodily function slowly deteriorates once symptoms manifest and will steadily worsen if left untreated. While its cause is unclear, ... [...More Info...] [...Related Items...] OR: [Wikipedia] [Google] [Baidu] |
