Solid organ and bone marrow transplantation have become more prominent in the field of inherited metabolic medicine over the past decade. Major advances in transplantation have allowed partial or complete cure in some inherited metabolic diseases for children and adults. Careful selection and planning are required for optimal survival. Longer term survivors now reveal more questions to be answered in terms of optimal monitoring and follow up. A multidisciplinary approach is essential in selecting and managing cases peri- and post-operatively to achieve good outcomes.
LEARNING OBJECTIVES
- Insights into the concepts of solid organ and bone marrow transplantation in children and adults with IMD.
- Understanding indications for different transplant types in specific IMD’s.
- Understanding current practices peri-and post-operatively in IMD and discuss existing guidelines.
- To enable participants to present difficult clinical scenarios.
- To enable participants and faculty to attend targeted workshops with case presentations and interactive discussions.
Institutes of main organisers - providers
- Evelina Children’s Hospital, St Thomas’ Hospital, London, United Kingdom
- Reference centre for inherited metabolic diseases, Jeanne de Flandre Hospital, Lille, France
- Royal Manchester Children’s Hospital, Manchester, United Kingdom
- Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, United Kingdom
Scientifc Organising Committee
- Roshni Vara, Evelina Children’s Hospital, St Thomas’ Hospital, London, United Kingdom
- Karine Mention, Reference centre for inherited metabolic diseases, Jeanne de Flandre Hospital, Lille, France
- Robert Wynn, Royal Manchester Children’s Hospital, Manchester, United Kingdom
- Robin Lachman, Charles Dent Metabolic Unit, National Hospital for Neurology and Neurosurgery, London, United Kingdom
Contact RRD Foundation
Cecilia Kellquist
Recordati Rare Diseases Fondation d'entreprise
Immeuble ‘Le Wilson’
70 avenue du Général de Gaulle
92800 PUTEAUX, France
Telephone : +33 1 47 73 86 11
Fax : +33 1 49 00 18 00
Email : ckellquist@rrd-foundation.org