Twelve years of experience with miglustat in the treatment of type 1 Gaucher disease: The Spanish ZAGAL project.
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L. Villalón | A. Acedo | A. Bárez | M. Andrade-Campos | E. Luño | P. Alfonso | M. Pocovi | P. Giraldo | V. Giner | O. Salamero | J. de la Serna | P. Irún | B. Medrano-Engay | M. Blanes | P. Latre | R. Franco | I. Roig | Á. Ibáñez | I. Loyola | K. Atutxa | V. Diaz-Morant | M. Fernández-Galán | Cristina Gil-Cortés | R. Hernández-Martin | J. Puerta | Ma Angeles Fernández-Galán
[1] D. Jayne,et al. SUMMARY OF PRODUCT CHARACTERISTICS , 2014 .
[2] C. Serratrice,et al. Switching from imiglucerase to miglustat for the treatment of French patients with Gaucher disease type 1: a case series , 2015, Journal of Medical Case Reports.
[3] B. Bembi,et al. Results from a 9‐year Intensive Safety Surveillance Scheme (IS3) in miglustat (Zavesca®)‐treated patients , 2015, Pharmacoepidemiology and drug safety.
[4] C. Hollak,et al. Miglustat therapy in type 1 Gaucher disease: clinical and safety outcomes in a multicenter retrospective cohort study. , 2013, Blood cells, molecules & diseases.
[5] M. Silkey,et al. Evaluation of miglustat as maintenance therapy after enzyme therapy in adults with stable type 1 Gaucher disease: a prospective, open-label non-inferiority study , 2012, Orphanet Journal of Rare Diseases.
[6] C. Hendriksz,et al. Gastrointestinal disturbances and their management in miglustat-treated patients , 2011, Journal of Inherited Metabolic Disease.
[7] P. Stein,et al. Osseous Manifestations of Adult Gaucher Disease in the Era of Enzyme Replacement Therapy , 2011, Medicine.
[8] C. Niederau,et al. Peripheral neuropathy in adult type 1 Gaucher disease: a 2-year prospective observational study. , 2010, Brain : a journal of neurology.
[9] A. Bárez,et al. Real-world clinical experience with long-term miglustat maintenance therapy in type 1 Gaucher disease: the ZAGAL project , 2009, Haematologica.
[10] B. Bembi,et al. Miglustat (Zavesca®) in type 1 Gaucher disease: 5‐year results of a post‐authorisation safety surveillance programme , 2009, Pharmacoepidemiology and drug safety.
[11] A. Mehta,et al. Goal-oriented therapy with miglustat in Gaucher disease. , 2009, Current medical research and opinion.
[12] S. van Weely,et al. Oral maintenance clinical trial with miglustat for type I Gaucher disease: switch from or combination with intravenous enzyme replacement. , 2007, Blood.
[13] Joel L Sussman,et al. Crystal structures of complexes of N-butyl- and N-nonyl-deoxynojirimycin bound to acid beta-glucosidase: insights into the mechanism of chemical chaperone action in Gaucher disease. , 2007, The Journal of biological chemistry.
[14] P. Alfonso,et al. Neurological evaluation of patients with Gaucher disease diagnosed as type 1 , 2007, Journal of Neurology, Neurosurgery, and Psychiatry.
[15] G. Pastores,et al. Effect of miglustat on bone disease in adults with type 1 Gaucher disease: a pooled analysis of three multinational, open-label studies. , 2007, Clinical therapeutics.
[16] P. Alfonso,et al. S-MRI score: A simple method for assessing bone marrow involvement in Gaucher disease. , 2007, European journal of radiology.
[17] O. Morand,et al. The pharmacokinetics and tissue distribution of the glucosylceramide synthase inhibitor miglustat in the rat , 2007, Xenobiotica; the fate of foreign compounds in biological systems.
[18] J. Yee,et al. The long-term international safety experience of imiglucerase therapy for Gaucher disease. , 2007, Molecular genetics and metabolism.
[19] A. Acedo,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. , 2006, Haematologica.
[20] S. Othman,et al. Comparison of bone mineral density with dual energy x-ray absorptiometry, quantitative ultrasound and single energy x-ray absorptiometry. , 2005, Saudi medical journal.
[21] J. Sancho,et al. Miglustat (NB-DNJ) works as a chaperone for mutated acid beta-glucosidase in cells transfected with several Gaucher disease mutations. , 2005, Blood cells, molecules & diseases.
[22] G. Pastores,et al. An open-label, noncomparative study of miglustat in type I Gaucher disease: efficacy and tolerability over 24 months of treatment. , 2005, Clinical therapeutics.
[23] R. Dwek,et al. Sustained therapeutic effects of oral miglustat (Zavesca, N-butyldeoxynojirimycin, OGT 918) in type I Gaucher disease , 2004, Journal of Inherited Metabolic Disease.
[24] G. Andria,et al. Therapeutic goals in the treatment of Gaucher disease. , 2004, Seminars in hematology.
[25] G. Andria,et al. The role of the iminosugar N-butyldeoxynojirimycin (miglustat) in the management of type I (non-neuronopathic) Gaucher disease: A position statement , 2003, Journal of Inherited Metabolic Disease.
[26] R. Dwek,et al. Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis , 2000, The Lancet.
[27] T. Cox,et al. 3 Gaucher's disease: clinical features and natural history , 1997 .
[28] T. Cox,et al. Gaucher's disease: clinical features and natural history. , 1997, Bailliere's clinical haematology.
[29] M. Itzchaki,et al. Effect of low-dose enzyme replacement therapy on bones in Gaucher disease patients with severe skeletal involvement. , 1996, Blood cells, molecules & diseases.
[30] J. Dambrosia,et al. Enzyme replacement therapy for Gaucher disease: skeletal responses to macrophage-targeted glucocerebrosidase. , 1995, Pediatrics.
[31] D. Balicki,et al. The clinical course of treated and untreated Gaucher disease. A study of 45 patients. , 1995, Blood cells, molecules & diseases.
[32] M. Horowitz,et al. Low-dose enzyme replacement therapy for Gaucher's disease: effects of age, sex, genotype, and clinical features on response to treatment. , 1994, The American journal of medicine.
[33] J. Dambrosia,et al. Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease. , 1991, The New England journal of medicine.