saccharomyces boulardii + miglustat

saccharomyces boulardii + miglustat is a combination treatment in which saccharomyces boulardii, a probiotic yeast, is co-administered with miglustat, an oral substrate reduction therapy indicated for certain lysosomal storage diseases such as type 1 Gaucher disease and Niemann-Pick disease type C. saccharomyces boulardii is used to supplement gut sucrase activity disrupted by miglustat's mechanism, with the goal of reducing miglustat-associated gastrointestinal side effects, particularly diarrhea. Miglustat acts as a competitive inhibitor of glucosylceramide synthase, thereby decreasing the synthesis and accumulation of glycosphingolipids, but can induce gastrointestinal adverse events by inhibiting intestinal disaccharidases[1][2]. saccharomyces boulardii provides a source of exogenous sucrase (invertase) to support sucrose digestion via a mechanism independent of human sucrase[1]. This combination has been investigated in a clinical trial as a means to improve gastrointestinal tolerability in individuals receiving miglustat[1].