You can find the slides of some presented talks below.
Download the full program here.
Friday, 4 November 2022
All in local time CDT (Dallas, TX, USA)
12.00 – 13.00 Registration
13.00 – 13.05 Welcome
Session 1: Update cohorts
13.05 - 13.20 Cohort update – ESMI (Jennifer Faber, DZNE Bonn, Germany)
13.20 - 13.35 The ARCA cohort: a global collaborative longitudinal cohort for advancing trial readiness in autosomal recessive cerebellar ataxias (Matthis Synofzik, University of Tübingen, Germany)
13.35 - 13.50 BIGPRO: the SCA3/MJD cohort from South Brazil. An update (Laura Jardim, Universidade Federal do Rio Grande do Sul, Brazil)
13.50 - 14.05 Cohort update – China (Hong Jiang, Central South University, China)
14.05 - 14.20 READISCA – An NIH-funded transatlantic observational study for SCA1 and SCA3 (Tetsuo Ashizawa, Houston Methodist Research Institute, USA)
14.20 - 14.30 Clinical phenotype and biomarkers in sporadic degenerative ataxia: longitudinal data from the SPORTAX registry (Thomas Klockgether, DZNE Bonn, Germany)
14.30 - 14.40 Mapping the natural history of SPG7: cross-sectional progression rates in 97 patients from the PROSPAX study (Rebecca Schüle, University of Heidelberg, Germany)
14.40 - 15.00 Data sharing success stories
ESMI (Thomas Klockgether, DZNE Bonn, Germany)
C-Path (Kael White, Ataxias Consortium at Critical Path Institute, USA)
15:00 - 15:45 Coffee break
Session 2: Patient-related outcomes
15.50- 16.10 KEYNOTE LECTURE: Activities of Daily Living (Christian Rummey, Clinical Data Science, Switzerland)
16.10- 16.25 Patient-focused outcomes in genetic ataxias: cross-sectional validity and longitudinal sensitivity to change of the FARS-ADL (Andreas Traschütz, University of Tübingen, Germany)
16.25 - 16.40 Determining clinical meaningful change of clinical and non-clinical outcomes: how can it be achieved? (Sophie Tezenas, Sorbonne University, France)
16.40- 16.50 Capturing clinical progression in multisystemic genetic ataxias: lessons from 1637 prospective assessments in patients with autosomal recessive or early-onset ataxia (Andreas Traschütz, University of Tübingen, Germany)
16.50 - 17.00 Temporal dynamics of the Scale for the Assessment and Rating of Ataxia (SARA) (Paul Moulaire, Sorbonne University, France)
17.00 - 17.40 Novel AGI activities
Work program for SARA modification (Thomas Klockgether, DZNE Bonn, Germany)
AGI Data Sharing Policy (Matthis Synofzik, University of Tübingen, Germany)
17.40 - 18.00 Group photo
18.00 - 19.30 Poster Session & Reception
20.00 AGI Steering Committee Meeting - invitation only -
Saturday, 5 November 2022
Session 3: Biomarkers
8.30 - 8.50 KEYNOTE LECTURE: Remote disease specific movement monitoring (Lisa Alcock, Newcastle University, UK)
8.50 - 9.20 Body worn sensors – remote monitoring in Ataxias: results from the US & Europe
A Score of Integrated Balance for Ataxia (SIBA) (James McNames, Portland State University, USA)
Towards ecologically valid biomarkers: real-life gait assessment capture subtle longitudinal and preataxic changes in cerebellar ataxia (Winfried Ilg, University of Tübingen, Germany)
9.20 - 9.35 Challenges and possibilities of automated speech monitoring in ataxia (Adam Vogel, University of Melbourne, Australia)
9.35 - 9.50 Blood levels of neurofilament light as trial biomarkers in degenerative ataxias: the examples of spinocerebellar ataxia type 1 (SCA1) and multiple system atrophy of cerebellar type (MSA-C) (Carlo Wilke, University of Tübingen, Germany)
9.50 - 10.05 Imaging biomarker recessive ataxias (Marcondes C Franca Junior, University of Campinas, Brazil)
10:05 - 10.15 Advanced multimodal MRI detects preataxic and early-stage alterations in SCA1 and SCA3 with high sensitivity (Gülin Öz, University of Minnesota, USA)
10.15 - 10.30 Subtle abnormalities of gait and posture in the prodromal stage of spinocerebellar ataxia type 2 (Yasmany González-Garcés, Centre for the Research and Rehabilitation of Hereditary Ataxias Holguín, Cuba)
10.30 - 11.00 Panel discussion: regulatory acceptance - presymptomatic stage (Stephanie Fradette, Biogen, USA; Michael Benatar, University of Miami, USA; Matthis Synofzik, University of Tübingen, Germany)
11.00 - 12.00 Coffee break
Session 4: Therapy
12:00 - 12:20 KEYNOTE LECTURE: Developing mutation-customized ASOs for ultra-rare and private ataxias (Willeke van Roon-Mom, Leiden University Medical Center, Netherlands)
12:20 - 12:40 KEYNOTE LECTURE: Genetic therapy approaches for neurodegenerative brain diseases and tackling the delivery challenge: the example of genetic FTD (Chris Shaw, King's College London, UK)
12:40 - 12:50 Increasing power of clinical trials in SCA1, 2, 3 and 6 with efficient designs and SARA scales (Emilien Petit, Sorbonne University, France)
12:50 - 13:00 Cerebellar transcranial direct current stimulation in spinocerebellar ataxia type 3: a randomized, double-blind, sham-controlled trial (Roderick Maas, Radboud University Medical Center, Netherlands)
13:00 - 13:30 Industry session
13.00 - 13.10 Development of VO659, a CAG repeat targeting ASO in development for PolyQ diseases (Scott Schobel, Vico Therapeutics)
13.10 - 13.20 AAV gene therapy for cardiac and central nervous system involvement in Friedreich’s Ataxia (Darin Falk, Lacerta Therapeutics, Inc.)
13.20 - 13.30 Improving clinical trial design using digital wearable technology in Friedreich ataxia (FA) and spinocerebellar ataxias (SCAs) 1, 2, 3, and 6 (Koene R.A. Van Dijk, Pfizer Inc.)
13.30 - 13.35 Wrap up (Matthis Synofzik, University of Tübingen, Germany)