Relevance to the EU scientific objectives

Why Us?
EUCLYD aims at establishing a unique research programme to tackle lysosomal storage diseases by putting together a consortium that will promote collaborative scientific interactions between the outstanding communities of basic and clinical investigators.

To improve human health, scientific discoveries must be translated into practical applications. EUCLYD proposes to take advantage of the basic scientific discoveries in lysosomal biology and in the basis of the pathogenetic mechanism due to substrate accumulation (WP1, WP2) to then progress towards the clinical level testing novel therapeutic approaches (WP3, WP4 and WP5) to eventually set the scenario to directly reach patient’s “bedside.”
The consortium and scientific programme was thought to allow the interaction between basic scientists and clinicians: basic science observations to provide clinicians developing therapeutic approaches with new tools for use in patients and for assessment of their impact, and clinical researchers to make novel observations regarding natural history, mutant-phenotype correlations on the nature and progression of disease that often stimulate basic investigations. The approach followed up by the consortium is in line with the priority stated in the Health work programme “Emphasis will be put on translational research for the development and validation of new therapies, methods for health promotion and prevention including promotion of child health, diagnostic tools and medical technologies”.

Why European?
Europe is a powerhouse for research in metabolic diseases. European centres are at leading edge of studies in lysosomal disorders and in the use of mouse models to elucidate the genetic bases of disease and to develop gene therapy approaches.

The EUCLYD consortium aims at developing a scientific network among outstanding communities of basic and clinical investigators in five European countries (Italy, Netherlands, UK, Sweden and Germany) to study various aspects of Lysosomal Storage Disorders (LSDs). The expertise brought together in the EUCLYD consortium (clinicians, basic scientists, human geneticists, gene therapy) is not found at the National level.
In addition, National funding agencies tend to avoid allocating funds to study rare genetic diseases, but rather prioritize and direct resources on diseases with a larger epidemiological impact such as cancer or diabetes.

No single European country could afford to finance a project of this dimension, which is why EC funding is so important. EU contribution to this project, integrated by Institutional funds, is indispensable for research into this important disorder group.