Yesterday I had my first visit with Dr. Susan Perlman, the head of the UCLA ataxia clinic. Dr. Perlman has been studying ataxia for 30 years and I am beyond lucky to have someone with her extensive knowledge right down the road from where I live.
The visit was incredibly positive. We discussed the state of research on ataxia and the various efforts to find ameliorative therapies as well as a cure itself. According to Dr. Perlman, we are 6 months out from a very promising clinical trial that will help with symptom progression. An actual cure may also be very close behind. One of the ideas for curing ataxia involves a concept called gene silencing. A company that recently changed its name from Isis Pharmaceuticals has been working on a gene silencing drug for years. In October 2015, a small set of patients with Huntington’s Disease (which is in the same set of diseases as ataxia) in Canada, Germany and the U.K. began a clinical trial of a gene silencing drug that effectively cured HD in animals. Next, with the FDA’s blessing, that drug will be tested on patients in the US with HD and ultimately ataxia. It’s incredibly good news. Hooray for ISIS!
The doctor also tested my current state of progression. Ataxia progression is measured on something called the SARA scale, which goes from 0 (asymptomatic) to 40 (the opposite of asymptomatic). I scored a 5.5. I’m not totally surprised because I was diagnosed very soon after symptom onset, so my number should be low. But the number is important for other reasons. Studies show that regular, intensive physiotherapy can reduce a SARA score by up to 5 points — roughly two years of degree progression. It effectively turns back the clock and, in my case, that clock could basically reset to 0. So, while we wait for a cure, I can give myself even more time to be mobile. I will have my first PT appointment when Corby and I return from Ireland.
Finally, we talked diet. It turns out Dr. Perlman was partially responsible for creating the ataxia diet I’ve started implementing. However, she was emphatic about one thing: no drinking. While a glass of red wine may have some helpful effects, that’s basically it. And so, for my health, I will say goodbye to all those lovely craft beers, bourbons, and ryes I have come to so enjoy. It’s a small price to pay in exchange for life.
So, there you have it. The battle lines have been drawn but the cavalry is coming. We will beat this yet.

Excellent news!!! Very encouraging!!!
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Well, this is positive news. The veil is lifting some.
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I promise to believe in something bigger than life itself if you are “cured” and your gene “silenced.” Thanks so much for this very uplifting news! Love you! Have a great time in Ireland!
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Like we discussed, we are living in the most amazing era in medical research and progress in history. On to a cure!
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Wonderfully promising. Guess Corb will be happy to drink your share of beer. Now to find the perfect red???
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This is wonderful news. You are amazing! Always have been!I can still remember you doing a dance performance as a little girl at Dorothy & Ed’s house, and your bravery and sense of self still shines through! Your attitude is so key! We can all learn from you! Robby & I send our love and are in the cheering section of your fan club! ….If you can have one Irish Whiskey while in Ireland, it may just be part of the cure…even though it is not on your new diet!
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Wonderful news. Have a wonderful time in Ireland and come back ready to do battle as I know you will
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Don’t give away those shoes just yet, missy! Love you. Tears of happiness today! xoxoxo
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