Patient-based Registry

Work Packages

The project’s activities are divided into eight work packages. With the strong support of patient organisations, the PATRAS registry will be established to provide data for advanced bioinformatic-assisted analysis of disease progression, variability and for the evaluation of new treatments. New disease models representing specific features of RASopathies will be generated. Disease models will be analysed to understand disease mechanism and to identify phenotypes and disease biomarkers. Novel technologies for drug screening platforms and for drug delivery for the brain will provide new effective and non-invasive treatments. Preclinical treatment trials will establish new effective therapies.

Communication activities will ensure optimal visibility, wide outreach to stakeholders and a solid base for results management.

For more information, please click on the graphic below
WP1Project management & scientific coordinationWP2PATRAS: advanced bioinformatics-assisted implementation and exploitationWP3 Generation and validation of disease modelsWP4Analysis of disease models to identify and evaluate "disease signatures"WP5 Phenotype-based drug screening and ASO developmentWP6New technologies for brain delivery of statins and ASOsWP7Evaluation of treatment successWP8Innovation management & patient engagement