Health: Destination 3: Tackling diseases and reducing disease burden
Development of new effective therapies for rare diseases

Scope: Proposals should aim to develop therapies for rare diseases with no approved therapeutic option. Proposals should focus on group(s) of rare diseases with commonalities, such as shared biological features, possibly within the same and/or across different medical areas within the rare diseases landscape.

Develop and utilise relevant preclinical models and/or innovative tools/technologies to verify molecular/cellular pathways/genes that can be therapeutically targeted, to increase the confidence in the target selection and/or to perform toxicity studies. When using disease models the applicants should describe how well the model replicates the pathology or the human condition.

Project budget: 8M (Total budget 60M); Deadline: 1 February 2022 (1st stage), 6 September 2022 (2nd stage). More

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