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European Working Group on GAUCHER Disease 7th EWGGD WORKSHOP St John’s College, Cambridge 18th – 22nd July 2006

Preliminary Programme Host Institution: University of Cambridge & Addenbrooke’s Hospital NHS Trust Workshop Organisers: T.M. Cox P.B. Deegan J.B. Grantham

www.ewggd.co.uk

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Welcome We are delighted to welcome you to the 7th International Workshop held under the auspices of the European Working Group on Gaucher Disease. This year the Workshop is being hosted by the team in the Department of Medicine of the University of Cambridge and Addenbrooke’s Hospital. We are very pleased to hold the meeting in one of the most beautiful of the riverside Cambridge Colleges – St. John’s. St. John’s College was founded in 1511 by Lady Margaret Beaufort, who was the mother of King Henry VIII. The EWGGD Workshop provides an opportunity for delegates to hear up-to-date reports on scientific discoveries, and to discuss their implications in relation to current practice. The meeting provides an opportunity for International Scientists and Medical Professionals to learn from each other and for patient representatives fully to participate. Despite its “orphan” designation, Gaucher’s disease has attracted immense competitive interest from industry. We thus also look forward to hearing about the latest pharmaceutical initiatives directly from those involved. We have excellent contributors who will address the many issues pertinent to the future of our patients. We trust that we will enjoy a memorable and informative Workshop and we greatly look forward to seeing you in July.

Tim Cox Professor of Medicine University of Cambridge Vice-Chairman EWGGD

Hans Aerts Professor of Biochemistry Amsterdam Medical Centre Chairman EWGGD

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E.W.G.G.D. Day 1 Tuesday 18th July 2006 A.M. 13:00 – 18:00 18:00 18:30 19:30

European Gaucher Alliance (EGA) meeting (in camera) Registration at St John’s College Reception Plenary Lecture: Professor Konrad Sandhoff Glycolipid catabolism and lysosomal membrane digestion Dinner in St. John’s College Dining Hall

Day 2 Wednesday 19th July 2006 9:00-13:00

Free Clinical Presentations Professor Ari Zimran ERT trial Professor Bryan Winchester Iminosugars as Antidiabetics, chaperones, enzyme Inhibitors Dr. Mario Maas Monitoring the skeleton in Gaucher disease

(Note: 3 x 30 min speakers & 30 min coffee) Two hours for free communications 8 x 15 min. (10 min + 5 min questions) 13:00 – 14:00

Lunch

14:00 – 14:30

Dr Anthony Futerman New Insights from the Structure of human glucocerebrosidase

14:30 – 17:00

Free Scientific Presentations (30 minutes)

(30 minutes)Two hours for free communications 8 x 15 min (10 min + 5 min questions) Social Programme alternatives Punting (1) Conducted tour Visit to cathedral city of Ely 19:00 for 19:30

Dinner in Chilford Hall, Linton

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Day 3 Thursday 20th July 2006 09:00 – 12:00 12.30 – 13.00 13:00 – 14:00

EGA/Gauchers Association/ECAP Sir Michael Rawlins Lunch

14:00 – 14:30 14:30 – 15:00 15:00 – 15:30 15:30 – 15.50 15.50 – 16:05 16:05 – 16:25

Science and Industry Genzyme (30) Actelion (30) Tea TKT Shire (20) Protalix (15) Healthcare at Home (20)

18:00 19:30

Plenary Lecture: Sir John Gurdon, FRS Nuclear re-programming in stem cells Gala dinner, St John’s College After dinner speaker: Professor Sir Keith Peters, FRS PMed Sci

Day 4 Friday 21st July 2006 09:00 – 13:00

Science Session Lectures: 1.TBA 2. Professor Chabas/Vilageliu/Grinberg Modifier genes 3. Dr. Greg Pastores How to bring lysosomal drugs to market and beyond

3 x 30 minute talks 30 minutes coffee 2 hours Scientific Poster Session (1st & last authors to attend) Poster questions, poster competition. 13:00 – 14:00

Lunch

14:00 – 17:00

Clinical Session 1. Dr Nadia Belmatoug Gaucher disease: Success and limitations of current Treatments 2. Dr Markus Ries (NIH) Treatment of Neuronopathic Gaucher disease

2 x 30 minute talks 2 hours for coffee and Clinical Poster session Poster questions, poster competition Social Programme alternatives Punting (2) Conducted tour Visit to Cathedral City of Ely 19:00

Dinner in St. John’s College

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Day 5 Saturday 22nd July 2006 09:00 – 13:00

1. Professor Hans Aerts TBA 2. Professor Milan Elleder Revisiting the pathology of Gaucher disease 3. Dr. R. Schiffman Neuronopathic Gaucher disease

Poster Prizes Announcement of next meeting 13:00

Close and Depart

Prize committee:

Dr P.K. Mistry, Professor Hans Aerts, Dr. Greg Pastores

Healthcare at Home - Poster Prize - Clinical Genzyme - Poster Prize - Scientific Actelion - Poster Prize for most original overall

Call for Abstracts

Deadline: Monday 12th May 2006 e-mail to: [email protected]

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Faculty List Professor Konrad Sandhoff Universitat Bonn, Germany Professor Ari Zimran Shaare Zedek Medical Centre, Jerusalem Professor Bryan Winchester Great Ormond Street Hospital, London, UK Dr Mario Maas Academic Medical Center, Amsterdam, Netherlands Dr Anthony Futerman Weizmann Institute, Israel Sir Michael Rawlins Chairman of N.I.C.E Sir John Gurdon, FRS Institute of Developmental Biology, Cambridge, UK Professor Sir Keith Peters, FRS Pmed Sci University of Cambridge, UK Professor Amparo Chabas University of Barcelona, Spain Professor Lluisa Vilageliu University of Barcelona, Spain Professor Daniel Grinberg University of Barcelona, Spain Dr Greg Pastores University Hospital of Manhattan, New York, USA Dr Nadia Belmatoug Hospital Beaujon, Paris, France Dr Markus Ries National Institute of Health, Bethesda, USA Professor Hans Aerts University of Amsterdam, Netherlands Professor Milan Elleder Charles University, Prague, Czech Republic Dr Raphael Schiffman National Institute of Health, Bethesda, USA Dr. Pramod K. Mistry Director, National Gaucher Disease Program Yale University, USA

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Registration Form Title

Surname:

First Name:

Job Title:

Hospital: Address for correspondence:

Postcode: Tel:

Fax:

Email: Meeting Options Please note that the registration fee covers all the conference dinners, lunches, teas and coffees, and delegate packs. Registration before 16th June including accommodation Registration after 16th June including accommodation Registration fee includes four nights accommodation

£600 ❏ £650 ❏

Registration before 16th June excluding accommodation Registration after 16th June excluding accommodation

£400 ❏ £450 ❏

I will attend the welcome dinner on Tuesday18th July I will attend the Conference dinner on Wednesday 19th July I will attend the Gala dinner on Thursday 20th July I will attend the Conference dinner on Friday 21st July

❏ ❏ ❏ ❏

Registration Total £ Include guest

❏ ❏ ❏ ❏

Please note there is single or twin bedded accommodation at St John’s College We would like to invite all accompanying persons to participate in the following activities. Wednesday 19th July Friday 21st July Punting £45 ❏ ❏ Conducted Tour of Cambridge £25 ❏ ❏ Visit to Cathedral City of Ely £45 ❏ ❏

Social Activities Total £

Payment Instructions Card Holders Name Visa/Mastercard/Amex/Maestro

Grand Total £

Card No Expiry Date

Issue No.

Security No.

Signature Cheques should be made payable to EWGGD Please send completed forms to: EWGGD 2006, 103a High Street, Stevenage, Herts SG1 3HR Tel +44 (0) 800 917 7405 Fax +44 (0) 1438 751520 E-mail [email protected] The closing date for registration is 7th July 2006.

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We would like to thank the following sponsors for their generous support Platinum Sponsorship

Gold Sponsorship

Bronze Sponsorship