Welcome to the website of the
European Gaucher Leadership Forum

Sponsored by Genzyme

Dear colleagues,

On behalf of the Scientific Steering Committee, I am delighted to invite you to the 2nd European Gaucher Leadership Forum which is to be held in London on the 1st and 2nd of October 2010.

The programme has been planned to provide a comprehensive update on recent advances in the pathophysiology, diagnosis and clinical management of Gaucher disease. Particular emphasis has been placed on covering practical clinical issues that have a direct impact on management of our patients.

Somewhat unusually, the Forum will be highly interactive and will include diverse sessions with different formats to facilitate discussion and active debate. An interactive case conference and poster tours have been included in the programme as well as plenary sessions with ample time to address questions to all the participants. The intention is to foster networking and exchange of experience among the Gaucher community, including clinicians, scientists and board members of the European Gaucher Alliance, representing the European patient associations.

As you might expect, time in the first session of the meeting has been allocated to discuss the clinical impact of the reduced supply of Cerezyme (imiglucerase). There will be an opportunity here to address the consequences of this crisis from every perspective. We have asked Genzyme to inform us about the production issues and the lessons that have been learnt in resolving them.

Skeletal manifestations are an important feature of Gaucher disease, and we will review the clinical evidence which examines the reduction in the risk of irreversible bone complications. In several presentations, we will review knowledge of the underlying mechanisms and course of bone disease, the role of imaging in monitoring and assessment and the best means to treat it. Time has also been allocated to Gaucher disease in children, allowing for discussion of the mechanisms underlying the different types, the clinical manifestations and optimum management.

A ‘Grand Round’ is a key feature of the programme and should provide the focus for informative plenary discussion about real-life management issues and the solution of clinical dilemmas. We will end the meeting with a look to the scientific future and include the new therapeutic options that are emerging, the underlying pathophysiology of the disease and the association between human glucocerebrosidase gene mutations and Parkinson’s disease.

On behalf of the international Scientific Steering Committee, I greatly look forward to welcoming you to London for this meeting and feel sure that you will find it a fulfilling and enjoyable professional experience.

Timothy M Cox
Professor of Medicine, University of Cambridge and Honorary Consultant Physician
Addenbrookes Hospital, Cambridge, UK

Scientific Steering Committee:
Dr Patrick Deegan
Metabolic Medicine Unit, Department of Medicine, University of Cambridge School of Clinical Medicine, Addenbrooke's Hospital, Cambridge, UK

Dr Carla Hollak
Academic Medical Center, Division of Endocrinology and Metabolism, Department of Internal Medicine, Amsterdam, The Netherlands.

Dr Anna Tylki-Szymańska
Clinic of Metabolic Diseases, Endocrinology and Diabetology, The Children's Memorial Health Institute, Warsaw, Poland

Dr Helen Michelakakis
Department of Enzymology and Cellular Function, Institute of Child Health, Aghia Sophia Children's Hospital, Athens, Greece