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16-17 June 2017
Warsaw, Poland

Gaucher Expert Summit 2017

Gaucher disease: Patient care across the generations

Meeting initiated, organized and funded by Shire International GmbH


Gaucher disease: Patient care across the generations

In addition to engaging plenary presentations, cutting edge keynote lectures, interactive panel discussions and frequent Q&As will provide a dynamic and open platform to explore current and future approaches to Gaucher disease management.


DAY 1 Programme: Friday 16 June

Time Presentation Faculty
08:30 Welcome to Poland Anna Tylki-Szymańska
08:40 Welcome from Shire Hartmann Wellhoefer
08:50 Chairs’ welcome Chairs
Scientific Session I – Addressing Gaucher disease in the early years

Session Chairs: Gregory Pastores & Anna Tylki-Szymańska
09:00 Diagnosing Gaucher disease in paediatric patients Anna Tylki-Szymańska
09:30 Clinical management of paediatric Gaucher disease Gregory Pastores
09:50 What have we learnt from GOS in paediatrics and pregnancy? Heather Lau
10:10 Panel Q&A All
10:40 Coffee
Scientific Session II – Biomarkers in future Gaucher disease management

Session Chairs: Maria Cappellini & Atul Mehta
11:00 Early diagnosis of Gaucher disease in adult patients Atul Mehta
11:20 Monitoring biomarkers in Gaucher disease: Is Lyso-Gb1 more than just a diagnostic tool? Jeff Szer
11:50 Is there a relationship between bone biomarkers and bone manifestations of Gaucher disease? Derralynn Hughes
12:10 Panel Q&A All
12:35 Lunch
13:35 Keynote lecture – An overview of lysosomes and lysosomal diseases Bryan Winchester
14:15 Poster Session
15:15 Coffee
Scientific Session III – Clinical trial design: It’s all in the detail

Session Chairs: Patrick Deegan & Ari Zimran
15:30 Challenging case studies: Audience briefing Maria Cappellini and Chairs
15:45 Keynote lecture – The importance of clinical trial design Mario Cozzolino
16:15 Overview of clinical trial designs in Gaucher disease Deborah Elstein
16:35 Interpretation of clinical trials in Gaucher disease Ari Zimran
16:55 Panel Q&A All
17:15 Summary and closing remarks Chairs
17:20 Close of Day 1

Next: Day Two Agenda

DAY 2 Programme: Saturday 17 June

Time Presentation
08:30 Chairs’ welcome Chairs
Scientific Session IV – Managing Gaucher disease over the long term

Session Chairs: Derralynn Hughes & Elena Lukina

08:35 Molecular and immunogenic profiles of ERTs Ari Zimran
09:00 Therapeutic management of Gaucher bone disease Nadia Belmatoug
09:25 Long-term clinical experience with ERT and SRT in adults with Gaucher disease Derralynn Hughes
09:50 What have we learnt from GOS? Patrick Deegan
10:10 Panel Q&A All
10:40 Coffee
11:00 Challenging case studies: Expert panel discussion Maria Cappellini and Chairs
12:00 Keynote lecture – Historical perspectives of Gaucher disease: From the past to the future Gregory Grabowski
12:40 Closing Remarks Chairs
12:50 Lunch and Departures

The Gaucher Expert Summit 2017 programme will examine the diagnosis of Gaucher disease in children and adults, the increasing importance of biomarkers in Gaucher disease, and therapeutic strategies for Gaucher disease management.
Meeting Chairs
Meet this year's chairs: Prof. Anna Tylki-Szymańska, Prof. Atul Mehta, and Prof. Ari Zimran.
The Hilton Warsaw Hotel and Convention Centre is easily accessible via car or public transportation.