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Burosumab
go.drugbank.com/drugs/DB14012ApprovedInvestigationalBurosumab (KRN23) is an entirely human monoclonal IgG1 antibody that binds excess fibroblast growth factor 23 (FGF23) and has been successfully tested in clinical trials in children with X-linked hypophosphatemic
Lurbinectedin
go.drugbank.com/drugs/DB12674ApprovedInvestigational[A214310] It is a derivative of the marine-derived agent ecteinascidin ([trabectedin]), an anticancer agent found in extracts of the tunicate _Ecteinascidia turbinata_, with the primary difference being
Categories: Cytochrome P-450 Substrates, P-glycoprotein inhibitorsOctreotide
go.drugbank.com/drugs/DB00104ApprovedInvestigational[L14501] In most cases, it results from an anterior pituitary growth hormone-releasing tumor.
Categories: P-glycoprotein substrates, Cytochrome P-450 CYP3A InhibitorsGadoquatrane
go.drugbank.com/drugs/DB33282Approved[L63814] Its distinguishing feature is its tetrameric structure carrying four chelated gadolinium ions with high relaxivity, which allows an adequately enhanced examination to be obtained while delivering
Trofinetide
go.drugbank.com/drugs/DB06045Approved[A258438] Trofinetide was approved by the FDA on March 10, 2023, for the treatment of Rett syndrome,[L45718,L45748] which is an X-linked neurodevelopmental disorder characterized by a range of cognitive
Categories: P-glycoprotein inhibitors, Cytochrome P-450 CYP3A InhibitorsCrinecerfont
go.drugbank.com/drugs/DB18518ApprovedInvestigational[A264818] Crinecerfont was approved by the FDA in December 2024 as an adjunct to glucocorticoid replacement in patients with CAH.[L51973,L51993]
Categories: Cytochrome P-450 Substrates, Cytochrome P-450 CYP2B6 SubstratesAGS-16C3F
go.drugbank.com/drugs/DB17551InvestigationalAGS 16C3F is an intravenously administered, anti-ectonucleotide pyrophosphatase/phosphodiesterase 3 (ENPP3) antibody-drug conjugate.
EGT-101
go.drugbank.com/drugs/DB17645ExperimentalEGT-101 is an adeno-associated viral vector serotype 9 (AAV9) vector-based gene therapy consisting of the human sulfamidase (SGSH) transgene.
ISIS 325568
go.drugbank.com/drugs/DB06099InvestigationalISIS 325568 is a generation 2.2 antisense drug that targets the glucagon receptor (GCGR). It was under investigation in clinical trial NCT00519727 (Safety Study of ISIS 325568 in Healthy Volunteers) for lowering blood glucose and helping...