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GsMtx-4
go.drugbank.com/drugs/DB17861InvestigationalGsMtx-4 is a peptide found in tarantula venom that inhibits mechanosensitive ion channel (MSC) activity.
Iptacopan
go.drugbank.com/drugs/DB16200ApprovedInvestigationalIptacopan is a small-molecule factor B inhibitor previously investigated as a potential treatment for the rare blood disease paroxysmal nocturnal hemoglobinuria (PNH) by inhibiting the complement factor … [A262566] Factor B is a positive regulator of the alternative complement pathway, where it activates C3 convertase and subsequently C5 convertase.
Synonyms: 4-((2S,4S)-4-ethoxy-1-((5-methoxy-7-methyl-1H-indol-4-yl)methyl)piperidin-2-yl) benzoic acid, BENZOIC ACID, 4-((2S,4S)-4-ETHOXY-1-((5-METHOXY-7-METHYL-1H-INDOL-4-YL)METHYL)-2-PIPERIDINYL)-Categories: Compounds used in a research, industrial, or household setting, Complement Factor B InhibitorKER-047
go.drugbank.com/drugs/DB19111InvestigationalKER-047 is under investigation in clinical trial NCT05927012 (A Study to Evaluate the Safety and Preliminary Efficacy of a Response-guided Dose Titration of KER-047 in the Treatment of Functional IDA (
Synonyms: QUINOLINE, 7-FLUORO-6-METHOXY-4-(6-(4-(4-METHYL-1-PIPERAZINYL)-1-PIPERIDINYL)PYRAZOLO(1,5-A)PYRIMIDIN-3-YL)-4-isothioureidobutyronitrile
go.drugbank.com/drugs/DB13010InvestigationalThioureidobutyronitrile has been used in trials studying the treatment of Solid Tumors and Ovarian cancer.
4-Iodopyrazole
go.drugbank.com/drugs/DB02721ExperimentalSynonyms: 4-IodopyrazoleCategories: Heterocyclic Compounds, 1-RingTividenofusp alfa
go.drugbank.com/drugs/DB17628ApprovedInvestigationalHunter syndrome is a rare, X-linked lysosomal storage disorder caused by a deficiency in the enzyme iduronate-2-sulfatase (IDS), which leads to the toxic accumulation of glycosaminoglycans (GAGs) throughout … Tividenofusp alfa is a first-in-class, brain-penetrant enzyme replacement therapy (ERT) indicated for the treatment of Mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome [L56097, L56099
Synonyms: - L-iduronate sulfate sulfatase, EC:3.1.6.13) pro-protein (1-525), fused via the peptide linker 526 GGGGS 530 to a human immunoglobulin G1 C-terminal Fc fragment (531-757) variant (L 544>A, L 545>A, T, 676>S, L 678>A, Y