Event name: 1st ARCA Global Conference
Date: 2-3 March 2020
Venue: Denver, USA
Information about the Conference
The 1st conference of the ARCA Global network, held in Denver, USA, March 2nd-3rd, 2020, aims to advance a global consortium for translational studies in autosomal-recessive ataxias (ARCAs), ARCA Global. ARCA Global is a dynamic, collaborative translational research initiative ultimately aiming to prepare and conduct harmonized multi-center treatment trials in genetically stratified ARCAs, thus allowing to translate the recent progress in ARCA genetics directly into stringent targeted treatment trials. It will integrate all main stakeholder groups including basic and clinician scientists, industry, and patient organizations. ARCA Global will aggregate ARCA patients and expertise from centers worldwide in close collaboration with patient organizations to facilitate all crucial translational steps on this path towards trial-readiness. It will:
- aggregate registry-inventoried ARCA cohorts from across the world
- perform genetic fingerprinting of unsolved ARCA patients, including defining novel ARCA genes and pathways
- lead standardized preclinical compound trials in ARCA model systems
- chart the natural history of ARCAs
- establish trial-ready clinical, imaging and molecular outcome measures for ARCAs
- implement first-in-human drug trials and prepare phase II and III studies in ARCAs.
There will be ample time allocated for both structured discussions led by peers and for informal discussion and social interactions to facilitate collaboration between all stakeholders and to systematically discuss criteria and infrastructure for global trial-readiness in ARCAs.
This 1st ARCA Global Conference will dovetail with the 8th Ataxia Investigators Meeting of the National Ataxia Foundation (NAF) to be held in Denver from March 3-6th, allowing interaction between ataxia investigators from two key ataxia meetings, as well as with patients/families/caregivers and industry partners.