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Gastrointestinal disturbances such as diarrhoea, flatulence and abdominal pain\/discomfort have consistently been reported as the most frequent adverse events associated with miglustat during clinical trials and in real\u2010world clinical practice settings. These adverse events are generally mild or moderate in severity, occurring mostly during the initial weeks of therapy. The mechanism underlying these gastrointestinal disturbances is the inhibition by miglustat of intestinal disaccharidase enzymes (mainly sucrase and maltase), leading to sub\u2010optimal hydrolysis of carbohydrates and subsequent osmotic diarrhoea and altered colonic fermentation. Transient decreases in body weight, which are often observed during initial miglustat therapy, are considered likely due to gastrointestinal carbohydrate malabsorption and associated negative caloric balance. While most cases of diarrhoea resolve spontaneously during continued miglustat therapy, diarrhoea also responds well to anti\u2010propulsive medications such as loperamide. Dietary modifications such as reduced consumption of dietary sucrose, maltose and lactose have been shown to improve the gastrointestinal tolerability of miglustat and reduce the magnitude of any changes in body weight, particularly if initiated at or before the start of therapy. Miglustat dose escalation at treatment initiation may also reduce gastrointestinal disturbances. This article discusses these aspects in detail, and provides practical recommendations on how to optimize the gastrointestinal tolerability of miglustat.<\/jats:p>","DOI":"10.1007\/s10545-011-9368-7","type":"journal-article","created":{"date-parts":[[2011,7,20]],"date-time":"2011-07-20T09:53:56Z","timestamp":1311155636000},"page":"991-1001","source":"Crossref","is-referenced-by-count":74,"title":["Gastrointestinal disturbances and their management in miglustat\u2010treated patients"],"prefix":"10.1002","volume":"34","author":[{"given":"Nadia","family":"Belmatoug","sequence":"first","affiliation":[{"name":"Reference Centre for Lysosomal Diseases Beaujon Hospital Clichy France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alberto","family":"Burlina","sequence":"additional","affiliation":[{"name":"Division of Metabolic Diseases University Hospital Padova Padova Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pilar","family":"Giraldo","sequence":"additional","affiliation":[{"name":"Miguel Servet Hospital CIBERER Zaragoza Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris J.","family":"Hendriksz","sequence":"additional","affiliation":[{"name":"Birmingham Children\u2032s Hospital NHS Foundation Trust Birmingham UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David J.","family":"Kuter","sequence":"additional","affiliation":[{"name":"Massachusetts General Hospital Boston MA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Eugen","family":"Mengel","sequence":"additional","affiliation":[{"name":"Villa Metabolica University of Mainz Mainz Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gregory M.","family":"Pastores","sequence":"additional","affiliation":[{"name":"Department of Neurology \u2010 Neurogenetics Unit, NYU Langone Medical Center 403 East 34th Street 2nd Floor New York NY 10016 USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2011,7,21]]},"reference":[{"key":"e_1_2_9_2_1","unstructured":"Actelion(2009)FDA advisory briefing book for miglustat (OGT 918 Zavesca\u00ae) in Niemann\u2010Pick type C disease. Retrieved 11th April 2011 fromhttp:\/\/www.fda.gov\/downloads\/AdvisoryCommittees\/CommitteesMeetingMaterials\/Drugs\/EndocrinologicandMetabolicDrugsAdvisoryCommittee\/UCM196758.pdf."},{"key":"e_1_2_9_3_1","unstructured":"Actelion(2010)Zavesca\u00ae (miglustat capsules) Prescribing Information. Available at:http:\/\/www.zavesca.com\/pdfs\/zavesca_pi.pdf. Accessed 9 June 2011."},{"key":"e_1_2_9_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0006-2952(99)00384-6"},{"key":"e_1_2_9_5_1","unstructured":"ChampionH RamaswamiU ImrieJ et al. 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