α-glucosidase, glycosyltransferase inhibitor. Inhibits galactosidase alpha (GLA), which interacts with SARS-CoV-2 Nsp14; analog of IHVR-19029, and antiviral in clinical trials. Gordon et al. bioRxiv, Mar 23 2020, https://doi.org/10.1101/2020.03.22.002386.
Biochemicals & reagents
1) Platt et al. (1994), N-butyldeoxynojirimycin is a novel inhibitor if glycolipid biosynthesis; J. Biol. Chem., 269 8362 2) Noel et al. (2008), Parallel improvement of sodium and chloride transport defects by miglustat (n-butyldeoxynojyrimicin) in cystic fibrosis; J. Pharmacol. Exp. Ther., 325 1016 3) Abian et al. (2011), 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., 8 2390 4) Serratrice et al. (2015), Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series; J. Med. Case Rep., 9 146 5) Karimzadeh (2013), Effects of miglustat on stabilization of neurological disorder in niemann-pick disease type C: Iranian pediatric case series; J. Child Neurol., 28 1599
Miglustat HCl (N-Butyldeoxynojirimycin) is an orally active ceramide-specific glycosyltransferase and α-glucosidase I and II inhibitor (1). This compound rescues trafficking-deficient F508del-CFTR in human airway epithelial cells via inhibition of ER α-glucosidases I and II (2), is a pharmacological chaperone for glucocerebrosidase degradation (3), is a clinically relevant agent for Gaucher disease type 1 (4) and it stabilizes neurological disorders in Niemann-Pick disease type C (5).