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Hrvatski
2013
First patient with RARB Mutation identified
2015
Research activities on RARB mutation started by the Michaud and Krezel Lab
2016
Establishment of disease models (ongoing)
Already funded
Patient registry and natural history studies (ongoing)
Costs: 95.000€
2020
Determination of disease mechanisms (ongoing)
Already funded but additional resources required: 195.000€
2021
Identification of biomarkers
Gene therapy approach vs. small molecule approach via drug repurposing
Costs: 1.030.000€
2022
Candidate Selection Point (CSP)
Preclinical safety trials
Costs: 1.200.000€
2023
Regulatory activities (Submission in USA and EU, IND / CTA)
Costs: 110.000€
2024
