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Newsletter January 2024
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- Launch of Ataxia Advisory Committee for Therapeutics
- Ataxia Global Conference 2023
- Relaunch of AGI WG 5 "Model System & Preclinical Trials"
- New Working Group on Patient Reported Outcomes
- Ataxia Webinars
- ICAR 2024 - Abstract Submission
- FDA Guidance on Rare Diseases
- Become an AGI Member or Partner
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Launch of Ataxia
Advisory Committee for Therapeutics
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AGI is pleased to announce that in 2024, the Ataxia Advisory Committee for Therapeutics (Ataxia-ACT) will have its first review session.
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The Ataxia-ACT is one of AGI’s trial readiness services. Its main objective is to review and provide objective advice on the planning and execution of preclinical and clinical drug development programs for (all forms of) ataxias.
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The Ataxia-ACT is a valuable resource and service to you. We provide transparent, unbiased, multi-stakeholder and multidisciplinary expert advice to optimize research, by either a) steering studies along a realistic and well-informed plan to clinical trial/later phase clinical activity or b) encouraging a change of direction or approach, to avoid failure further down the line.
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Ataxia Global Conference 2023
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Ataxia Global Conference 2023 on Best Endpoints in Ataxia took place on 15-16 November, online.
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Four AGI Working groups worked on taking ataxia trial-readiness to the next level: from outcomes to endpoints. Best endpoint scenarios were presented for Digital-motor gait/balance measures, Patient-Reported Outcomes, MRI, and NfL.
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We had amazing 288 registrations, four great sessions and fruitful and enthusiastic discussions. Thank you all for participation and your work towards Ataxia trial readiness!
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- Each AGI WG: focus the work on gaps in moving from outcomes to endpoints
- Publish most advanced AGI WG work as distinct paper
- Overarching state-of-the art AGI viewpoint paper across 5 outcome domains (t.b.d. further)
- Overarching (cross-WG) AGI focus project “Anchoring of Outcomes/Biomarkers in Patient-Meaningful Variables”
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Relaunch of WG 5
"Model Systems & Preclinical trials"
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AGI started a relaunch of our working group 5 “Model systems & preclinical trials”. The new WG Leads are:
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- Magda Santana (Center for Neurosciences and Cell Biology, University of Coimbra, Portugal)
- Jeannette Hübener-Schmid (University Hospital Tübingen, Germany)
- Ronald Buijsen (LUMC, Leiden, the Netherlands)
- Thorsten Schmidt (University Hospital Tübingen, Germany)
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Are you interested in the goals described below and have experience in the topic?
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📢You are welcome to join the working group!
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For AGI members or employees of an AGI partner organization:
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The main goal of this working group is the standardization of preclinical research that is essential to establish trial-readiness for ataxias.
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These will include the following topics, but are not limited to:
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- 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
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To achieve our goals, we will:
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Milestone 1: Create an inventory of existing models and protocols;
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Milestone 2: Establish a database for sharing the collected information;
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Milestone 3: Develop standardized protocols and guidelines based on collected information;
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Milestone 4: Develop training material and tools and conduct training sessions for ataxia preclinical researchers.
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New Working Group on
Patient Reported Outcomes
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The AGI is setting up a new Working Group on Patient Reported Outcomes Measurements (PROM). The aim of the WG is to define a set of data including a graded PROMs catalogue that will serve as the standard for future sharing of clinical data and joint clinical studies.
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The first pre-work results were presented at the Ataxia Global Conference by the core group. The set-up of the WG will be initiated by the AGI office in the near future.
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iScience: AGI YII Webinar Series on Hot Topics
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Explore Your Future in Ataxia Research
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Speakers: Filippo Santorelli (Pisa, Italy), Puneet Opal (Chicago, USA) - WG2 Molecular biomarkers & biosampling
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Tuesday, 30 January 2024, 5-6 pm CET
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ICAR 2024 - Abstract Submission
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FDA Guidance on Rare Diseases
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FDA invites you to comment on the guidance here
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Become an AGI Member or Partner
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Become an AGI member or partner!
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The application for membership (as individual) and partnership (as organization) of the AGI is available on our website. More information about membership and partnership can be found in our charter.
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As a member, you can join the AGI working groups, nominate and vote for new members for the AGI Steering Committee, and attend our business meetings.
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