Arimoclomol citrate is a heat shock protein amplifier potentially useful for the treatment of Niemann-Pick disease type C (NP-C). Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones.
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Name | Arimoclomol citrate |
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Iupac Chemical Name | 3-Pyridinecarboximidoyl chloride, N-((2R)-2-hydroxy-3-(1-piperidinyl)propoxy)-, 1-oxide, 2-hydroxy-1,2,3-propanetricarboxylate (1:1) |
Synonyms | Arimoclomol citrate; BRX-220; BRX 220; BRX220; BRX-345; BRX 345; BRX345; |
Molecular Formula | C20H28ClN3O10 |
Molecular Weight | 505.90 |
Smile | O[C@@H](CON=C(Cl)c1ccc[n+]([O-])c1)CN2CCCCC2.OC(=O)CC(O)(CC(=O)O)C(=O)O |
InChiKey | XSENLDLUMVYRET-BTQNPOSSSA-N |
InChi | InChI=1S/C14H20ClN3O3.C6H8O7/c15-14(12-5-4-8-18(20)9-12)16-21-11-13(19)10-17-6-2-1-3-7-17;7-3(8)1-6(13,5(11)12)2-4(9)10/h4-5,8-9,13,19H,1-3,6-7,10-11H2;13H,1-2H2,(H,7,8)(H,9,10)(H,11,12)/t13-;/m1./s1 |
CAS Number | 368860-21-3 |
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Formulation | Off-white solid |
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Purity | 98% Min. |
Storage | Dry, dark and at 0 - 4℃ for short term (days to weeks) or -20℃ for long term (months to years). |
Solubility | Soluble in DMSO |
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Shipping Condition | Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs. |
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Clinical study |
1: McDermott CJ. Clinical trials in amyotrophic lateral sclerosis. Curr Opin Neurol. 2019 Oct;32(5):758-763. doi: 10.1097/WCO.0000000000000731. PubMed PMID: 31335338.
2: Hettinghouse A, Liu CJ. Brain-penetrant heat shock protein amplifier arimoclomol enhances GCase activity in in vitro Gaucher disease models. EBioMedicine. 2018 Dec;38:7-8. doi: 10.1016/j.ebiom.2018.11.052. Epub 2018 Dec 3. PubMed PMID: 30522930; PubMed Central PMCID: PMC6306393.
3: Fog CK, Zago P, Malini E, Solanko LM, Peruzzo P, Bornaes C, Magnoni R, Mehmedbasic A, Petersen NHT, Bembi B, Aerts JFMG, Dardis A, Kirkegaard T. The heat shock protein amplifier arimoclomol improves refolding, maturation and lysosomal activity of glucocerebrosidase. EBioMedicine. 2018 Dec;38:142-153. doi: 10.1016/j.ebiom.2018.11.037. Epub 2018 Nov 27. PubMed PMID: 30497978; PubMed Central PMCID: PMC6306395.