#5 Model systems & preclinical trials

Working Group 5
WG Name Model systems & preclinical trials
WG Leads Magda Santana (Coimbra, Portugal)
Ronald Buijsen (Leiden, the Netherlands)
Jeannette Hübener-Schmid (Tübingen, Germany)
Thorsten Schmidt (Tübingen, Germany)
WG Objectives The main goal of this working group is the standardization of preclinical research that is essential to establish trial-readiness for ataxias.

These will include the following topics, but are not limited to:

  • Animal models (e.g. breeding, genotyping, behavior, histology, and biochemistry analysis)
  • Cellular models, including patients derived neuronal cultures (e.g. culturing, differentiation, and functional assays)
  • Pre-clinical biomarkers


To achieve our goals, we will:

Milestone 1: Create an inventory of existing models and protocols;

Milestone 2: Establish a database for sharing the collected information;

Milestone 3: Develop standardized protocols and guidelines based on collected information;

Milestone 4: Develop training material and tools and conduct training sessions for ataxia preclinical researchers.

If you are interested in participating in working group 5, please follow this link.