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Huntington's Disease Youth Organization

Is there feedback from the gene silencing trial?

HDYO has more information about HD available for young people, parents and professionals on our site:

Q. Hi I have recently tested gene positive I was wondering if there any feedback from the gene silencing trials trying to remain hopeful

Sheree, Young Adult, Australia

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A. Dear Sheree,

Thanks for your email and for your great question. You may know that gene silencing has been successfully used in animals who carry the HD gene. The first ever trial in humans has just started. I heard from the person in charge of that trial that they had just started with the first patient about 3 weeks ago. The main purpose of this trial is to check to see if it’s safe to give to humans, so at this stage there are going to be around 40 participants involved and they will be started on the drug one at a time. So you can see it’s being done in a cautious way.

If the treatment is safe and without serious side effects, the next step will be to do a much bigger trial to test whether or not the treatment is effective (as well as safe). So the good news is that this research is happening right now. However, it will take time for the results to come through and to allow for sensible planning of follow-up trials. You can get more information about this trial on HDBuzz.

Best wishes


Last updated: March 04, 2017 08:02