Program

Monday 22 November 2021

Session 1: Genetics & New Targets/Pathways

14.00 - 14.05 CET: Welcome

14.05 - 14.35: Keynote lecture: "Challenges and opportunities in ataxia genomics”
Stephan Züchner (University of Miami)

14.35 - 14.55: AGI Working Groups - WG6: Next-generation ataxia genomics and platforms
Matthis Synofzik (University of Tübingen)

14.55 - 15.15: AGI Projects - Genetic modifiers in CANVAS and other repeat expansion disorders
Henry Houlden (University College London)

15.15 - 15.35: AGI Projects - Rapid generation of knock-out stem cell models of ARCAs
Ricardo Schnekenberg (University of Oxford)

Session 2: AGI Business Meeting (members and partners only)

15.50 - 16.05: Introduction
Thomas Klockgether (Chair of the AGI Steering Committee, University of Bonn)

16.05 - 16.25: Business presentation
Holm Graessner (Head of the AGI office, University of Tübingen)

16.25 - 16.35: Role of patient organisations in AGI
Sue Hagen (National Ataxia Foundation)
Julie Greenfield (Ataxia UK)

16.35 - 16.45: Trial readiness services
Birte Zurek (AGI office, University of Tübingen)

16.45 - 16.55: Collaboration with CPTA
Terina Martinez (CPTA)

16.55 - 17.25: Next actions and discussion
Annemarie Post (AGI office, University of Tübingen)

Tuesday 23 November 2021

Session 3: Biomarkers and natural history

19.30 - 19.35 CET: Welcome

19.35 - 20.00: Keynote lecture: "Regulatory aspects in neurodegenerative disorders"
Karl Broich (Federal Institute for Drugs and Medical Devices, Germany)

20.00 - 20.17: AGI Working Groups - WG3: MR biomarkers
Gülin Öz (University of Minnesota)

20.17 - 20.29: AGI Projects - Identification and characterization of biomarkers from blood plasma and CSF of spinocerebellar ataxia-1 (SCA1) patients
Puneet Opal (Northwestern University)

20.29 - 20.41: AGI Projects - PROSPAX: an integrated multimodal progression chart in spastic ataxias (ARSACS, SPG7)
Rebecca Schüle (University of Tübingen)

20.41 - 20.53: PAHAN survey on the care for ataxias in the Americas and the Caribbean
Laura Bannach Jardim (Federal University of Rio Grande do Sul)

Session 4: Therapy

21.00 - 21.30: Keynote lecture: "ASO therapies in Huntington's disease"
Fabricio Pio (University of British Columbia)

21.30 - 22.00: Keynote lecture - Gene therapy for polyQ SCAs
Nathalie Cartier (INSERM)

22.00 - 22.15: AGI Working Groups - WG1: Clinical outcomes
Thomas Klockgether (University of Bonn)

22.15 - 22.30: AGI Projects - CACNA1A-related ataxia: functional characterization of clinically severe variants for drug repurposing
Enrico Bertini (Bambino Gesù Children’s Hospital)