Results from a 9-year Intensive Safety Surveillance Scheme (IS3 ) in miglustat (Zavesca® )-treated patients

Author:

Brand Monika1,Muller Audrey1,Alsop Jonathan2,van Schaik Ivo N.3,Bembi Bruno4,Hughes Derralynn5

Affiliation:

1. Actelion Pharmaceuticals Ltd; Allschwil Switzerland

2. Numerus Ltd; Wokingham UK

3. Academic Medical Center; Amsterdam the Netherlands

4. Regional Coordinator Centre for Rare Diseases; Azienda Ospedaliera Santa Maria della Misericordia di Udine; Udine Italy

5. Royal Free and University College Medical School; London UK

Funder

Actelion Pharmaceuticals

Publisher

Wiley

Subject

Pharmacology (medical),Epidemiology

Reference15 articles.

1. Actelion Miglustat prescribing information 2014 http://www.zavesca.com/pdf/ZAVESCA-Full-Prescribing-Information.pdf

2. Actelion Miglustat (Zavesca) summary of product characteristics 2014 http://www.eudrapharm.eu/eudrapharm/productDetailsAction.do

3. Disease and patient characteristics in NP-C patients: findings from an international disease registry;Patterson;Orphanet J Rare Dis,2013

4. Prevalence of lysosomal storage disorders;Meikle;JAMA,1999

5. Novel oral treatment of Gaucher's disease with N-butyldeoxynojirimycin (OGT 918) to decrease substrate biosynthesis;Cox;Lancet,2000

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