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The CEPT was set up last year for the benefit of Gaucher disease patients at high-risk for the development of progressive disease or complications and without access to alternative treatments during the period of Cerezyme shortage CEPT will be reinstated now due to prolonged shortage of Cerezyme supplies and based on a recommendation of the European Medicine Agency (EMA).
Why do we need a pan European Umbrella group?
The working together of Gaucher patient groups has already lead to consistency in treatment of certain manifestations of Gaucher Disease throughout Europe. Inspired by this and by their successes in achieving humanitarian aid for the most severe patients in Eastern Europe member countries of the EGA havegrasped the challenge of collaboration for the further advantage of Gaucher Patients.
Although the most common Lysosomal Storage Disorder Gauchers Disease is very rare with most European countries having less than 300 patients.
Patient Groups have worked closely together to share their experiences and to facilitate access to treatment. Scientists and Clinicians throughout Europe and the rest of the world increasingly collaborate both in research and in the development of strategies for the management of the disease.
Pharmaceutical companies are Global and clinical trials are frequently multi-centred with treatments being approved for use throughout Europe.
Patient Groups in Europe cannot work in isolation and their alliance to formalisethe EGA provides a clear European voice of representation and opportunity for advancement.