U.S. Department of Health & Human Services Divider Arrow National Institutes of Health Divider Arrow NCATS

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Showing 221 - 230 of 623 results

Status:
Designated
Source:
FDA ORPHAN DRUG:800720
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
FDA ORPHAN DRUG:166203
Source URL:

Class (Stereo):
CHEMICAL (ACHIRAL)

TH9402 is a dibrominated rhodamine derivative and potent photosensitizer and useful in photodynamic therapy. TH9402 preferentially localizes in mitochondria and when exposed to 500- to 600-nm wavelength visible light delivered through the Theralux device, it becomes highly cytotoxic through oxidative damage. TH9402 eradicates multiple myeloma (MM) and BC cell lines, while sparing more than 50% of normal pluripotential blood stem cells from healthy volunteers. TH9402 offers an excellent potential as an ex vivo photodynamic purging agent for autologous transplantation in multiple myeloma and breast cancer treatment. TH9402 photodynamic-cell-therapy process conditions recognized as safe for normal human haematopoietic stem cells and progenitors demonstrated the efficacy of the purging procedure on various leukaemias.
Status:
Designated
Source:
FDA ORPHAN DRUG:693119
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
EU-Orphan Drug:EU/3/14/1375
Source URL:

Class (Stereo):
CHEMICAL (ABSOLUTE)

Status:
Designated
Source:
FDA ORPHAN DRUG:618517
Source URL:

Class (Stereo):
CHEMICAL (RACEMIC)

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.

Showing 221 - 230 of 623 results