• Project summary
• WP 1 Understanding misfolding, amyloid and toxicity
• WP 2 Dissection of the amyloid deposits
• WP3 Genetic analyses
• WP4 Animal models
• WP5 Development and resolution
• WP6 Development of diagnostic procedures and clinical parameters
• WP7 Development of treatments

WP7 Development of treatments

Important advancements in the treatment of systemic amyloidosis have been achieved in the last decade. However, not all patients can benefit from treatment and for some types of amyloidosis the therapy is still largely unsatisfactory. Therefore, there is an urgent need for more effective and less toxic therapeutic tools.

EURAMY represents a unique opportunity to transfer new knowledge, technologies and therapeutic agents developed in the experimental laboratories to the bed-side, for the care of patients, through a collaborative effort between basic scientists, pathologists and clinicians. The development of new drugs is based on the knowledge acquired on the basic mechanisms of amyloid formation and tissue toxicity. Drugs active in vitro will then be tested in cell cultures and animal models for toxicity and efficiency. Clinical researches shall optimize the efficiency of drugs already on trial, and test the safety and efficiency of novel drugs. One of the objectives is the establishment of a European network for the care of systemic amyloidoses, in collaboration with patients’ associations, which represents a unique resource for testing novel agents and for disseminating the knowledge on these rare diseases.