Developing Patient Organisations Throughout The World

Similar groups were forming in Europe and around the world and in 1992, the Genzyme Corporation sponsored an international symposium for clinicians, scientists and patients’ support groups in Amsterdam. Representatives of patients’ support groups from many countries were able to establish a network of contacts that still flourishes. Both the German and Italian patients’ support groups were established following the symposium, spurred on by the longer established groups.

In each country, patient support groups for Gaucher disease were started by a few patients and their families seeking information, and many of these founders remain active in their groups to this day. Although a few are from the medical profession, the majority come from diverse backgrounds – lawyers to writers, entrepreneurs to office workers. A doctor whose son has Gaucher disease also started off a patient group with the same lack of knowledge which was all too common. However, all those involved soon became experts in this narrow field of lysosomal storage diseases, acquiring the knowledge and experience from a variety of sources and contacts which turned them into health information and healthcare professionals.  They have been able to provide valuable information to families and participate in discussions with doctors, scientists and health authority officials on both a local and national level. This knowledge, and their personal drive led them to negotiate with high level representatives from the pharmaceutical industry to obtain factual information, devoid of marketing bias, which could be verified by clinical experts.