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Restrict the search for
vitamin a
to a specific field?
Status:
Designated
Source:
EU-Orphan Drug:EU/3/15/1481(POSITIVE)
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
BN-82451 belongs to a family of small molecules designated as multitargeting or hybrid molecules. BN-82451 is orally active, has good central nervous system penetration, and elicits potent neuronal protection and antiinflammatory properties. BN-82451 acts via three major pathways involved in neuronal death: excito-toxicity, oxidative stress, and inflammation, and is also a mitochondrial protective agent. Because BN-82451 is a multitargeting agent, each of its specific sites of action has been extensively evaluated, namely, neuronal excitotoxicity (sodium channel blocker), oxida-tive stress (antioxidant), neuroinflammation (cyclooxygenase inhibitor), and mitochondrialdysfunction (mitochondria-protective properties). BN-82451 was found to exert a significant protection in experimental animal models mimicking aspects of cerebral ischemia, Parkinson disease, Huntington disease, and more particularly amyotrophic lateral sclerosis. BN-82451 is in phase II clinical trials by Ipsen Pharma for the treatment of Huntington’s disease. In 2015, orphan drug designation was assigned in the U.S. to the compound for the indication.
Status:
Designated
Source:
FDA ORPHAN DRUG:18887
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
B-Trisaccharide belongs to the class of organic compounds known as fatty acyl glycosides of mono- and disaccharides. B-Trisaccharide in blood group B is excreted when an individual is given Galactose. The B-trisaccharide was the only oligosaccharide detected in plasma after galactose administration to a blood-group-B secretor individual. The structure of the branched blood group B trisaccharide alone has been studied both by X-ray crystallography and by NMR.
Status:
Designated
Source:
FDA ORPHAN DRUG:18086
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
A-TRISACCHARIDE (or Blood group A trisaccharide), a core antigen fragment in ABO blood group system. It was found to be a major urinary carbohydrate depending on diet and blood type.
Status:
Designated
Source:
FDA ORPHAN DRUG:452214
Source URL:
Class (Stereo):
CHEMICAL (MIXED)
Status:
Designated
Source:
FDA ORPHAN DRUG:312010
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Epitalon (amino acid sequence Ala-Glu-Asp-Gly) is a synthetic peptide that has been suggested to induce the activation of ribosomal genes, decondensation of pericentromeric structural heterochromatin and the release of genes repressed due to the age-related condensation of euchromatic chromosome regions. The researchers who generated Epitalon also reported an inhibitory effect on the development of spontaneous tumors in mice, and claimed that it increases neuronal activity and can be used in the treatment of Retinitis Pigmentosa. The peptide is still under pre‐clinical and clinical development and thus has not been approved for any therapeutic and/or prophylactic use by any government health authority in the USA or Europe.
Status:
Designated
Source:
FDA ORPHAN DRUG:390013
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Targets:
Bromopyruvate is an halogenated analogue of pyruvic acid known as an alkylating agent reacting with thiol groups of many proteins. Bromopyruvate exerts anticancer action. It is based on the impairment of energy metabolism of tumor cells by inhibiting enzymes in the glycolysis pathway (hexokinase II, glyceraldehyde 3-phosphate dehydrogenase, phosphoglycerate kinase) and the oxidative phosphorylation (succinate dehydrogenase). Bromopyruvate induces endoplasmic reticulum stress, inhibits global protein synthesis further contributing to cancer cell death. Treatment with bromopyruvate has been administered in several cancer type models both in vitro and in vivo, either alone or in combination with other anticancer therapeutic approaches. These studies clearly demonstrate bromopyruvate broad action against multiple cancer types. This compound has also antifungal and antiparasitic activity.
Status:
Designated
Source:
FDA ORPHAN DRUG:935523
Source URL:
Class (Stereo):
CHEMICAL (ACHIRAL)
Conditions:
Luminol (5-amino-2,3-dihydro-1,4-phthalazinedione) is a yellow-colored crystalline solid powder and soluble in most polar organic solvents, but insoluble in water. An alkaline solution of luminol oxidized by oxidizing agents exhibits chemiluminescence. Luminol was first synthesized by Schmitz in 1902, the chemiluminescence property of luminol was first discovered by Albrecht in 1928. Luminol is one of the most widely used chemiluminescent compounds because of its availability and low cost. Luminol-based methods are used in environmental monitoring as biosensors, in the pharmaceutical industry for cellular localization and as biological tracers, and in reporter gene-based assays and several other immunoassays.
Status:
Designated
Source:
FDA ORPHAN DRUG:230206
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Gossypol is a substance that is found in the cotton plant. It is removed from the seeds and used for medicine. Gossypol is effective as a nonhormonal male contraceptive; however, it has been documented to have irreversible effects on male fertility. Gossypol is reported to exhibit antioxidant, anticancer, antivirus, antiparasitic, and antimicrobial properties and lower plasma cholesterol. Nausea, emesis, anorexia, diarrhea, altered taste sensation, small intestine obstruction, and fatigue have been recorded in clinical trials as adverse reactions. Large amounts of gossypol can decrease potassium levels in the body. Low potassium levels can increase the side effects of digoxin.
Status:
Designated
Source:
FDA ORPHAN DRUG:95896
Source URL:
Class (Stereo):
CHEMICAL (ABSOLUTE)
Targets:
Conditions:
Etiocholanedione is a natural metabolite of dehydroepiandrosterone with potent antiobesity activity. Etiocholanedione has been identified as a metabolite of an altered androgen metabolism that eventually leads hepatocellular carcinoma to impaired hormone responsiveness in human. Etiocholanedione has been identified as a metabolite of 17alpha-hydroxyprogesterone in some patients affected by congenital adrenal hyperplasia, although it doesn't appear to account for the masculinization observed in congenital hyperplasia.
