Report of the MPS 2011 International Congress in Geneva

This meeting brought together over 125 participants including about sixty scientists from international research organizations, universities, health institutions, private industry. Among the scientists, 27 were from countries of the European Union (including 12 from France), 27 were from Switzerland and 8 from the United States, Canada and Australia. Thirty-six families also participated to the congress. Countries represented by the families included; France (9), Switzerland (4), Spain (4), Turkey (4), Russia (3), Portugal (2), Bosnia and Herzegovina (2) , United States (2), Argentina (1), Italy (1), Great Britain (1), Poland (1), Ireland (1), and Belarus (1) . The families included three adults with MPS IVA and VI, and seven children aged 6 to 10 years suffering from Sanfilippo disease. The children participated to a social program organised by the Foundation Cap Loisirs, Geneva.

The aim of the conference was to provide an update on the latest developments for MPS by bringing together researchers and clinicians as well as parents of children with MPS. MPS is a group of rare genetic diseases which includes Sanfilippo disease. Considering the sad fact that there is no cure for the Sanfilippo syndrome one of the goals of this first conference was to stimulate research on this disease.

The conference opened with welcoming speeches by Dr Elizabeth Mason of the World Health Organization (WHO), Prof. Jacques-André Romand, Geneva State General Surgeon, Dr. Loredana D'Amato Sizonenko, Head of the Orphanet platform in Switzerland, and Mr. Dominique Dunant, President, Carigest SA. The conference consisted of 24 oral presentations and 5 posters. Keynote speakers of the scientific program included Prof. John Hopwood, from the Women's and Children's Hospital, Adelaide, Australia; Dr Patrick Haslett, Shire Human Genetic Therapies, Massachusetts, United States; Prof. Marc Tardieu, Bicetre Hospital, Paris and Prof. Lena Kjellen, Uppsala University, Sweden. Twenty other speakers completed the program including researchers from the Universities of Graz, Gdansk, Padova, Orleans, Paris, Barcelona, Manchester, Montreal, Houston, Cincinnati, Columbus, EPFL Lausanne, as well as representatives from the private industry including Lysogene, Zacharon and Orphazyme together with the French Myopathy Association, the Blackswan Foundation and the Gebert Rüf Stiftung both based in Switzerland.

The film produced by Marie-Fleur Stalder entitled "Children of Sanfilippo" was also screened during the session for families. The conference ended with a closing speech by Esther Neiditsch Prigioni, President of ProRaris, Alliance for Rare Diseases, Switzerland.

According to Prof. Marc Tardieu "This conference, which brought together almost all the specialists of the field, has been very helpful." The conference was an opportunity to exchange and share information between researchers in the same field in the presence of families of patients on current developments in treatment options for different types of MPS. Importantly, this meeting led to collaborations between researchers. Two research teams from the United States and Britain decided to collaborate on research work for a treatment for Sanfilippo disease and three companies will study the possibility to combine their work in the same direction.

The Sanfilippo Foundation Switzerland was approached by another association to organize a future congress to provide an update on research in the field. New research projects were also submitted to the Sanfilippo Foundation to apply for financial support.

Families invited spoke of a "Conference of Hope" and came away feeling less alone in the face of illness. Research into MPS disorders is advancing and producing results, it is therefore reasonable to expect that an effective treatment can be found in the near future.

THE SPONSORS OF THE EVENT