When combined with medicine to reduce blood pressure (antihypertensive drugs), it is not clear if treatments that lower homocysteine can help prevent a stroke in some people. Hypotheses have been offered to address the failure of homocysteine-lowering therapies to reduce cardiovascular events. When folic acid is given as a supplement, it may increase the build-up of arterial plaque. A second hypothesis involves the methylation of genes in vascular cells by folic acid and vitamin B12, which may also accelerate plaque growth. Finally, altered methylation may catalyse l-arginine to asymmetric dimethylarginine, which is known to increase the risk of vascular disease. See also
Homocystinuria
Kilmer S. McCully

References


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People with type I Gaucher have a defect in the enzyme called glucocerebrosidase (also known as acid β-glucosidase). Glucocerebrosidase is an enzyme, and its function is to convert glucocerebroside (also known as glucosylceramide) into ceramide and glucose. When this enzyme doesnt work, glucocerebroside accumulates, which in turn causes liver and spleen enlargement, changes in the bone marrow and blood, and bone disease. It functions as a competitive and reversible inhibitor of the enzyme  glucosylceramide synthase,  the  initial  enzyme  in  a  series  of  reactions  which  results  in  the  synthesis  of  most glycosphingolipids.Earlier treatments on the market (imiglucerase (approved in 1995), velaglucerase (approved in 2010), taliglucerase alfa (Elelyso) (approved in 2012)) are enzyme replacement therapy - they are functioning versions of the enzyme that doesnt work. Miglustat works differently - it prevents the formation of the substance that builds up when the enzyme doesnt work; this is called substrate reduction therapy. Chemistry
Miglustat is an iminosugar, a synthetic analogue of D-glucose and a white to off-white crystalline solid that has a bitter taste. Society and culture
Legal status
Miglustat has been approved in the EU, Canada, and Japan for treating progressive neurological complications in people with Niemann–Pick disease, type C (NPC). Miglustat has not received approval for this indication outside of these countries and use for this disease, including the US, is off-label.