Description
Miglustat HCl (210110-90-0) is an orally active ceramide-specific glycosyltransferase and α-glucosidase I and II inhibitor.1 Rescues trafficking-deficient F508del-CFTR in human airway epithelial cells via inhibition of ER α-glucosidases I and II.2 Pharmacological chaperone for glucocerebrosidase degradation.3 Clinically useful agent for Gaucher disease type 1.4 Stabilizes neurological disorders in Niemann-Pick disease type C.5
Uses
An inhibitor of a-glucosidase 1 as well as being an inhibitor of HIV cytopathicity. N-Butyldeoxynojirimycin has recently been used to retard lipid storage in the central nervous system of an animal m
odel of Tay-Sachs disease.
1-Deoxynojirimycin derivatives as selective inhibitors of glucosylceramide metabolism in man.
References
[1] F. PLATT. N-butyldeoxynojirimycin is a novel inhibitor of glycolipid biosynthesis.[J]. The Journal of Biological Chemistry, 1994, 106 1: 8362-8365. DOI:
10.1016/s0021-9258(17)37202-2[2] SABRINA NOËL. Parallel improvement of sodium and chloride transport defects by miglustat (n-butyldeoxynojyrimicin) in cystic fibrosis epithelial cells.[J]. Journal of Pharmacology and Experimental Therapeutics, 2008, 325 3: 1016-1023. DOI:
10.1124/jpet.107.135582[3] OLGA ABIAN*. Therapeutic Strategies for Gaucher Disease: Miglustat (NB-DNJ) as a Pharmacological Chaperone for Glucocerebrosidase and the Different Thermostability of Velaglucerase Alfa and Imiglucerase[J]. Molecular Pharmaceutics, 2011, 8 6: 2390-2397. DOI:
10.1021/mp200313e[4] CHRISTINE SERRATRICE Jacques S Laure Swiader. Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series.[J]. Journal of Medical Case Reports, 2015, 9: 146. DOI:
10.1186/s13256-015-0617-5[5] PARVANEH KARIMZADEH. Effects of miglustat on stabilization of neurological disorder in niemann-pick disease type C: Iranian pediatric case series.[J]. Journal of Child Neurology, 2013, 28 12: 1599-1606. DOI:
10.1177/0883073812464526
