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[CAS NO. 210110-90-0] Miglustathydrochloride
| Ships within | Stock | Price | Qty | Total |
Please click "REQUEST A QUOTE" button if there is no stock, or you need other sizes or custom synthesis.
| Catalog: | HY-17020A |
| Brand: | MCE |
| CAS: | 210110-90-0 |
Overview
| MDL | - |
|---|---|
| Molecular Weight | 255.74 |
| Molecular Formula | C10H22ClNO4 |
| SMILES | O[C@H]1[C@H](O)[C@@H](CO)N(CCCC)C[C@@H]1O.[H]Cl |
2 Publications Citing Use of MCE
Summary
Clinical Trial
| NCT Number | Sponsor | Condition | Start Date | Phase |
|---|---|---|---|---|
| NCT05174039 | Beyond Batten Disease Foundation|Theranexus |
Batten Disease
|
February 2, 2022 | Phase 1|Phase 2 |
| NCT00945347 | Cliniques universitaires Saint-Luc- Université Catholique de Louvain |
Cystic Fibrosis
|
July 2009 | Phase 2 |
| NCT00517153 | Actelion |
Niemann-Pick Type C Disease
|
January 2002 | Phase 2 |
| NCT00742092 | Actelion |
Cystic Fibrosis
|
August 2008 | Phase 2 |
| NCT04768166 | IRCCS Fondazione Stella Maris |
Hereditary Spastic Paraparesis
|
June 15, 2021 | Phase 2 |
| NCT03822013 | Tehran University of Medical Sciences|Mashhad University of Medical Sciences|Kashan University of Medical Sciences |
GM2 Gangliosidosis|Supportive Care
|
January 14, 2019 | Phase 3 |
| NCT02325362 | Assistance Publique - Hôpitaux de Paris|Actelion|CRCM (Centres de Ressources et de Compétences de la Mucoviscidose) |
Cystic Fibrosis
|
March 17, 2015 | Phase 2|Phase 3 |
| NCT00041535 | National Institute of Neurological Disorders and Stroke (NINDS)|National Institutes of Health Clinical Center (CC) |
Gaucher Disease
|
July 5, 2002 | Phase 2 |
| NCT01760564 | National Taiwan University Hospital |
Niemann-Pick Disease Type C
|
January 2008 | Phase 3 |
| NCT02030015 | University of Minnesota|Rare Diseases Clinical Research Network|National Center for Advancing Translational Sciences (NCATS)|National Institute of Neurological Disorders and Stroke (NINDS)|National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)|Lysosomal Disease Network |
GM1 Gangliosidoses|GM2 Gangliosidoses|Tay-Sachs Disease|Sandhoff Disease
|
December 22, 2015 | Phase 4 |
| NCT02520934 | National Taiwan University Hospital|Actelion |
Gaucher Disease
|
July 2015 | Not Applicable |
| NCT04808505 | Amicus Therapeutics |
Glycogen Storage Disease Type II Infantile Onset
|
August 2022 | Phase 3 |
| NCT00537602 | Actelion |
Cystic Fibrosis
|
November 2007 | Phase 2 |
| NCT03910621 | Actelion |
Niemann-Pick Disease, Type C
|
April 2, 2020 | Phase 4 |
| NCT01451879 | University of Florida |
Pompe Disease
|
October 2008 | |
| NCT00316498 | National Eye Institute (NEI)|National Institutes of Health Clinical Center (CC) |
Niemann Pick Diseases
|
October 17, 2002 | Phase 1 |
| NCT00319046 | Actelion |
Gaucher Disease Type 1
|
February 1, 2006 | Phase 3 |
| NCT00418847 | The Hospital for Sick Children|Actelion |
Gangliosidoses GM2
|
July 2004 | Phase 2 |
| NCT00194649 | University of Washington|Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) |
Contraception
|
June 2005 | Phase 4 |
| NCT03911505 | Amicus Therapeutics |
Pompe Disease (Late-onset)
|
February 13, 2020 | Phase 3 |
| NCT00672022 | Children´s National Research Institute|Actelion |
GM2 Gangliosidoses|Tay-Sachs|Sandhoff Disease
|
July 2004 | Phase 3 |
Appearance
Solid
Shipping
Room temperature in continental US; may vary elsewhere.
Storage
-20°C, stored under nitrogen
* In solvent : -80°C, 6 months; -20°C, 1 month (stored under nitrogen)
Solvent & Solubility
DMSO : 65 mg/mL ( 254.16 mM ; Need ultrasonic)
H 2 O : ≥ 34 mg/mL ( 132.95 mM )
* "≥" means soluble, but saturation unknown.
Stock Solutions
| Concentration Solvent Mass | 1 mg | 5 mg | 10 mg |
|---|
| 1 mM | 3.9102 mL | 19.5511 mL | 39.1022 mL |
| 5 mM | 0.7820 mL | 3.9102 mL | 7.8204 mL |
| 10 mM | 0.3910 mL | 1.9551 mL | 3.9102 mL |
-
1.
Add each solvent one by one: PBS
Solubility: 100 mg/mL (391.02 mM); Clear solution; Need ultrasonic
-
2.
-
3.
Add each solvent one by one: 10% DMSO >> 90% (20% SBE-β-CD in saline)
Solubility: ≥ 3.25 mg/mL (12.71 mM); Clear solution
-
4.
Add each solvent one by one: 10% DMSO >> 90% corn oil
Solubility: ≥ 3.25 mg/mL (12.71 mM); Clear solution
References
- [1] Abian, O., et al., Therapeutic strategies for Gaucher disease: miglustat (NB-DNJ) as a pharmacological chaperone for glucocerebrosidase and the different thermostability of velaglucerase alfa and imiglucerase. Mol Pharm, 2011. 8(6): p. 2390-7.
- [2] van Giersbergen, P.L. and J. Dingemanse, Influence of food intake on the pharmacokinetics of miglustat, an inhibitor of glucosylceramide synthase. J Clin Pharmacol, 2007. 47(10): p. 1277-82.
- [3] Miglustat. Mother To Baby | Fact Sheet, Organization of Teratology Information Specialists (OTIS), 1 April 2021.
- [4] Neal J Weinreb, et al. Guidance on the use of miglustat for treating patients with type 1 Gaucher disease. Am J Hematol. 2005 Nov;80(3):223-9.
Synonyms
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