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C9orf72 Gene Therapy Targeting Trial UK - Phase 1

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    C9orf72 Gene Therapy Targeting Trial UK - Phase 1

    This is a big deal for some of our (favourite ) Forum Members.

    Phase 1 of its clinical trial started this week, so very early days, but it’ll be very interesting to see the trial results when published.

    https://mndresearch.blog/2019/09/19/...ial-in-the-uk/

    Love Ellie.
    ​Diagnosed 03/2007. Sporadic Definite ALS/MND Spinal (hand) Onset.
    Eye gaze user - No working limbs - No speech - Feeding tube - Overnight NIV.


    #2
    This is early days but this along with the biogen SOD1 Trial are in my mind the key trials ongoing in the world today.

    Rock on!

    Comment


      #3
      Thanks Ellie. I read all about it previously but forgot. Appreciate the reminder. I read it again. There's other research going on but no other to my knowledge for C9ORF72 in the world. I get the ALS news letters.

      I hope that the participants of this trial stay safe and the scientists can learn from it. Obviously I hope that it's the first of 3 associated trials and that it turns out to be a useful treatment but I'm keeping my feet firmly planted. Lynne
      ALS diagnosed November 2017, limb onset. For the 4 yrs previously I was losing my ballance.
      I'm staying positive and taking each day as it comes.

      Comment


        #4
        I hope to get on the Phase 1 trial at the National Hospital for Neurology in central London. I was phoned by one of the doctors there yesterday. I plan to visit them in the next few weeks for an informal chat. This is indeed a big deal for those of us with the C9orf72 mutation. They are running 6 cohorts with 8 people in each. Cohort 1 is given a very low dose of drug, Cohort 2 gets a higher dose and so on. The cohorts run in series. The aim, as I understand it, is to assess people's tolerance to the drug.

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          #5
          Good luck John. I hope that it doesn't make you feel ill. Take care. Lynne
          ALS diagnosed November 2017, limb onset. For the 4 yrs previously I was losing my ballance.
          I'm staying positive and taking each day as it comes.

          Comment


            #6
            Fingers cossed that you get on the trial John and it goes well.

            Love Ellie.
            ​Diagnosed 03/2007. Sporadic Definite ALS/MND Spinal (hand) Onset.
            Eye gaze user - No working limbs - No speech - Feeding tube - Overnight NIV.

            Comment


              #7
              Exciting times! I hope you are, indeed, taken onto the trial, John.
              Dina

              Trying to keep positive, but not always managing.

              Comment


                #8
                Thank you, all. I'll post updates.

                Comment

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