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Ataxia and I: clinical trials, let’s talk about it!

Hi friends!

Once again, I am moved by the nice welcome you give to this blog and I really want to have the time to answer you all … Without being able to do that, I will try to direct my words towards answering everyone. I hope that will be enough; if I have a role of “light carrier” to assume as wrote a reader, as much play it to the end!

So today, I’m going to talk about upcoming clinical trials in gene therapy and I’ll write about how they make me feel. A hot, hot subject for people with AF. Because, indeed, one of the most promising for us, in Canada, is that of Dr. Tremblay and his team of researchers at Laval University. They managed, by gene therapy, to “cure” transgenic mice with FA. We are therefore at the door of testing this method on humans, but it remains stubbornly closed due to lack of solid financial support.

Frankly, my opinion on this discovery is quite contradictory – like a bit of everything in my life but… In other words, I am optimistic, yes, but above all realistic and terribly lucid. So, i encourage you to make your own opinion!

In short, my position is simple: on the one hand, this cure would not be miraculous, but the faulty gene causing Friedreich Ataxia would be corrected, finally allowing the expression of the frataxin protein by the cells. These so-called cells would then have a second life … Except the nerve cells that do not regenerate once atrophied. And FA is an essentially neurological disease … It is then impossible to predict how sick bodies would recover; after all, to participate in any clinical trial is to offer yourself as a guinea pig in the name of science. … So, I hate the term «miracle healing” that clinical trials evoke for many ataxics. That some refuse to live the present moment by being constantly turned towards the future, to what promises (or not) clinical trials, it makes me cringe, I can’t help it!

On the other hand, for those whose symptoms are still slight, for future generations or for society in general, the impact will be entirely positive. I am looking forward to Dr. Tremblay’s research developments: if this therapy in gene therapy works for FA, it is applicable for any other ataxias and genetic disease !!

Not to mention the beneficial effects that a clinical trial would have on the entourage of a patient with ataxia; it would be a welcome whiff of hope for them!

In fact, what I think of the medical progress surrounding my condition can be summed up as “too much hope, kills hope.” You must live and hope that if it happens, it’s good! Despite the illness, life is surprising and deserves to be lived! 😉

For our next appointment, unless there is another request, I think I’ll go for a look at home care services for people with reduced autonomy.

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