European network of paediatric research-European Medicines Agency meeting on rare gastrointestinal and liver diseases

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TitleEuropean network of paediatric research-European Medicines Agency meeting on rare gastrointestinal and liver diseases
Date08/12/2015 - 08/12/2015
LocationEuropean Medicines Agency, London, UK
SummaryThe European network of paediatric research (Enpr-EMA) is organising a one-day meeting. The morning session is dedicated to the Paediatric European Digestive Diseases Clinical Research Network (PEDDCReN) to present to industry representatives their planned initiative to prospectively identify the incidence and prevalence of rare and very rare diseases in the field of paediatric gastroenterology and hepatology by establishing a European registry for these rare diseases and to discuss with industry how best to use this to work for developing research in these unique groups of patients. The afternoon session is organised together with the European Cystic Fibrosis Society and will focus on gastrointestinal outcome measures for clinical trials with new medicines targeting the basic defect in cystic fibrosis, so-called CFTR modulators.

All documents

Name Language First published Last updated
Report on the European network of paediatric research-European Medicines Agency workshop on gastrointestinal (GI) outcome measures to evaluate CFTR modulators for the treatment of cystic fibrosis (CF) (English only) 2016-03-16  
Agenda - European network of paediatric research-European Medicines Agency meeting: rare gastrointestinal and liver diseases initiative (morning session) (English only) 2015-11-19  
Agenda - European network of paediatric research-European Medicines Agency meeting: workshop on gastrointestinal (GI) outcome measures to evaluate CFTR modulators for the treatment of cystic fibrosis (CF) (afternoon session) (English only) 2015-11-19  
Presentation - Intestinal Pseudo-obstruction (Gut Motility Disorders) (Nikhil Thapar) (English only) 2016-01-19  
Presentation - Chronic Intractable Diarrhoea of infancy (Nick Croft) (English only) 2016-01-19  
Presentation - Rare liver diseases (Ulrich Baumann) (English only) 2016-01-19  
Presentation - PIBD Cancer & Mortality (Lissy de Ridder) (English only) 2016-01-19  
Presentation - Why are rare diseases of interest to industry (Brihad Abhyankar) (English only) 2016-01-19  
Presentation - Rare diseases, European and UK approaches: setting the scene (Julian Thomas) (English only) 2016-01-19  
Presentation - How the EMA EU support treatment development in rare diseases (Laura Fregonese) (English only) 2016-01-19  
Presentation - Paediatric European Digestive Diseases Clinical Research Network Project (Nick Croft) (English only) 2016-01-19  
Presentation - Overview of CFTR Modifier Pipeline (Tim Lee) (English only) 2016-01-19  
Presentation - Potential of GI outcome measures in CFTR modulation (Frank Bodewes) (English only) 2016-01-26  
Presentation - The role of intestinal organoid function for evaluation of CFTR modulators (Jeffrey Beekman) (English only) 2016-01-26  

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enprema@ema.europa.eu