Research update 7/24

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 Dr. Gomez, Hannah Casey, and his Team are making great progress with the electronic questionnaire. They have submitted their plan for this to their Institutional Review Board for approval. It can take some time to satisfy all reviewer questions but it is a good milestone to have submitted their plan to them. This electronic questionnaire will include people with SCA6 or SCA27B, a newly identified form of ataxia with symptoms similar to SCA6. This is smart because it allows for comparison of the comprehensive responses for each group to allow for a better understanding of the similarities and differences between these 2 ataxia types. We will let you know when it becomes available. 


I know many of you are interested in hearing about the SCA6 research in Dr. GOmez's lab. I see a new publication titled: "Digital Measures of Postural Sway Quantify Balance Deficits in Spinocerebellar Ataxia"


I know we all are looking forward to an effective clinical trial for SCA6 and better measures of outcome or whether the trial intervention s working or not helps attract the pharmaceutical companies to consider opening such trials. It seems the anti-sense oligonucleotide approach to therapy has not panned out the way we hoped but that a better approach might be gene therapy as a single infusion prevention of symptom development or even CRISPR has potential. I know in the meanwhile many of you are working hard with physical therapy and in other ways to stay as healthy as possible. Keep it up! I truly appreciate all of your positive energy and willingness to help one another. If anyone is wanting the opportunity to participate in a weekly social, sometimes fun, sometimes, educational, sometimes serious, but usually begins with a joke group, consider joining Susan Stile's AR&DG. (sstiles12@gmail.com). You don't have to go every week  and she maintains an agenda so you can go to meetings that interest you most. I

If Dr. Gomez's questionnaire is ready in a month, I might ask him to present about it at our next BOF Q&A session. If not, I will start thinking about organizing the next one in August or September anyway. As always, please send me any questions you might have about SCA6 and I will start collating them for him ahead of time. 

My very warmest wishes to all of you, but STAY COOL!

Terry

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