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National Ataxia Foundation

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ICAR Thursday Afternoon: Discoveries for Potential Ataxia Treatments

Written by Dr. Amy Smith-Dijak
Edited by Celeste Suart

Let’s talk treatment. Thursday afternoon starts off with some debates about testing and administering new therapies. Later attendees will hear about the latest clinical trials for ataxia therapies. Work with patients? There is also a workshop on the SARA scale.

Thursday afternoon will be mostly focused on treatment. The afternoon will start off with a series of interactive debates about testing and administering new therapies. Debate topics will include the role of gene therapy in the future of ataxia treatment, the best way to decide whether a clinical trial was successful, and what kind of animal models are useful for testing certain types of therapies. Each debate will be led by two experts on the topic who will give their opinions and moderate comments from the audience. While these debates may not come to a conclusive decision, attendees will hear new perspectives and perhaps continue these discussions beyond the official confines of the conference.

The debate will be followed by two concurrent sessions: a seminar on emerging therapies, and a workshop on the SARA scale. During the seminar, researchers will present the results of trials they’ve been running on potential therapies for ataxias. These trials cover a wide range of therapies, from small-molecule drugs to physical training. Presenting all of these trials together will provide a snapshot of the state of research on the treatment of ataxia. For clinicians, this will be an opportunity to find out about promising therapies that their patients may benefit from now or in the future. For researchers, this will be a chance to assess which therapies are moving successfully from the laboratory to the clinic.

Finally, the workshop on the SARA scale aims to train clinicians and researchers to score ataxia severity. This is useful both for clinicians following individual patients and for researchers who are trying to see what kind of impact their treatment is having. The SARA scale gives patients a score based on a series of tests of their movement control. While this sounds simple the scoring can be somewhat subjective, which leaves room for inconsistency between scorers. This workshop will help attendees to be more consistent in their scoring, which will make it easier to discuss patients and compare clinical trial results.

In all, the Thursday afternoon session is an opportunity for everyone working on ataxia therapies to get together and compare notes. These kinds of events help the community figure out the best approaches to research and the most promising avenues of investigation. This session will be a great time to discuss treatments for ataxias and how best to study them.

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