Journal of Inherited Metabolic Disease

, Volume 35, Issue 6, pp 949–954 | Cite as

Miglustat-induced intestinal carbohydrate malabsorption is due to the inhibition of α-glucosidases, but not β-galactosidases

Rapid Communication


Miglustat is an oral medication that has approved indication for type I Gaucher disease and Niemann pick disease type C. Usually treatment with Miglustat is associated with occurrence of gastrointestinal side effects similar to carbohydrate maldigestion symptoms. Here, we studied the direct influence of Miglustat on the enzymatic function of the major disaccharidases of the intestinal epithelium. Our findings show that an immediate effect of Miglustat is its interference with carbohydrate digestion in the intestinal lumen via reversible inhibition of disaccharidases that cleave α-glycosidically linked carbohydrates. Higher non physiological concentrations of Miglustat can partly affect lactase activity. We further show that the inhibition of the disaccharidases function by Miglustat is mainly competitive and does not occur via alteration of the enzyme folding.


Gauche Disease Intestinal Lumen Brush Border Membrane Lactase Miglustat 









lactase-phlorizin hydrolase


brush border membrane



A Ph.D. scholarship provided by the German Academic Exchange Service (DAAD) to M.A. We thank Heshmat Akbari-Borhani for critical discussion.

Conflict of interest

This study has been supported by Actelion Pharmaceuticals GmbH.


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Copyright information

© SSIEM and Springer 2012

Authors and Affiliations

  1. 1.Department of Physiological ChemistryUniversity of Veterinary Medicine HannoverHannoverGermany

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