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    • #14474

      Pfizer is going to collaborate with REGENXBIO to use NAV AAV technology to develop new gene therapies to treat Friedreich’s ataxia. Read the full story here.

      What do you think of this news?

    • #14480
      Matt Lafleur

      This is very hopeful it seems!

    • #14555

      My wife’s uncle is an orthopedic surgeon and he looked into this for us and from what he could tell it is very promising. For years he said the cure for FA wasn’t possible because of FA being in the genes. This new breakthrough actually can change all that. And from what he could tell it won’t matter how far along your progression is for it to help considerably. Obviously it’s not going to reverse damages done by FA or things related to FA like scoliosis, damage to heart muscle, diabetes, etc. Although diabetes could be overcome just by being able to become more active, as long as your pancreas is still able to produce a little insulin on its own.
      He said this is the most promising treatment for FA as well as other genetic disorders(like Cf, CP, etc.) he has looked into so far. So it’s not just going to help FAers. Though they are focusing on primarily FA. From what he said it looks like it’s not just to lessen symptoms like most treatments that has come before but be able to actually reverse most if not all of the FA.
      VERY promising he said emphatically. He couldn’t tell how long it might be for sure though.

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