Gastrointestinal disturbances and their management in miglustat-treated patients

  title={Gastrointestinal disturbances and their management in miglustat-treated patients},
  author={Nadia Belmatoug and Alberto B. Burlina and Pilar Giraldo and Christian J. Hendriksz and David J Kuter and Eugen Mengel and Gregory McCarthy Pastores},
  journal={Journal of Inherited Metabolic Disease},
Miglustat (Zavesca®) is approved for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease (GD1) for whom enzyme replacement therapy is unsuitable, and for the treatment of progressive neurological manifestations in adult and paediatric patients with Niemann-Pick disease type C (NP-C). Gastrointestinal disturbances such as diarrhoea, flatulence and abdominal pain/discomfort have consistently been reported as the most frequent adverse events associated with miglustat… CONTINUE READING


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