In type 1 Gaucher disease
Pastores G.M. et al. Effect of miglustat on bone disease in adult patients with type 1 Gaucher disease: a pooled analysis of three multinational Open-label studies. Clinical Therapeutics. 29: 1645-53; 2007.
Elstein D. et al. Oral maintenance clinical trial with miglustat for type 1 Gaucher disease: switch from or combination with intravenous enzyme replacement. Blood. 110: 2296-2301; 2007.
Giraldo P. et al. Short-term effect of miglustat in every day clinical use in treatment-naïve or previously treated patients with type 1 Gaucher’s disease. Haematologica. 91:125-8; 2006.
Elstein D. et al. Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type 1 Gaucher disease. J Inherit Metab Dis 27: 757-66; 2004.
In Niemann-Pick type C disease
Patterson M.C., Vecchio D., Prady H., Abel L., Wraith J.E. Miglustat for treatment of Niemann-Pick C disease: a randomised controlled study. Lancet Neurol 6,765-772; 2007.
Patterson M.C., Vecchio D., Prady H., Abel L., Wraith J.E. Miglustat for treatment of Niemann-Pick C disease: results of 24 month's treatment. Proceedings of 57th Annual meeting of the American Society of Human Genetics, 2007; abstract # 2253.
Pineda M, Wraith JE, Sedel F, et al. Miglustat in patients with Niemann-Pick type C disease (NPC): a multicentre retrospective survey. Journal of Inherited Metabolic Disease 31(Suppl 1) 98; 2008.
