SMA News Forums Forums Treatments and Research Zolgensma (onasemnogene abeparvovec) I Feel Gratitude in the Wake of Zolgensma

  • I Feel Gratitude in the Wake of Zolgensma

    Posted by brianna-albers on June 4, 2019 at 3:00 pm

    Happy Tuesday, everyone! I’m sure most of you are aware of Zolgensma by now, the new gene therapy for SMA. I went back and forth on whether to write a column about it, and in the end, I decided to. I’ve thought a lot about “cures” and how they might affect me, so I wasn’t surprised to realize that, the more I thought about Zolgensma, the less excited I was.

    Don’t get me wrong: Zolgensma is an amazing breakthrough, and I’m looking forward to where things go from here. I’ve just always had a complicated relationship with “cures” and treatments, maybe because I know myself well enough to realize that if I let myself imagine life post-Zolgensma—a life where I can walk, tie my own shoes, hold a job—I’ll quickly grow dissatisfied with the life I have. As much as I want to be “cured,” the pessimist in me is stubborn, and the realist wants to live the life I have, imperfect but real, to the fullest.

    Is this something you can relate to? How do you approach treatments like Zolgensma and Spinraza?

    brianna-albers replied 5 years ago 3 Members · 4 Replies
  • 4 Replies
  • ryan-berhar

    Member
    June 4, 2019 at 8:35 pm

    I actually think you’re right on with this. Zolgensma is an absolutely enormous breakthrough, but first, it’s not approved for adults yet, and second, I don’t want my contentment relying on a cure.

  • deann-r

    Member
    June 5, 2019 at 5:27 pm

    This reminds me when several years ago, before even a glimmer of a treatment, my sister asked if there was a cure if I would get it. I told her that even if there was a cure my body has been so affected I’d never be “normal.” My fused spine doesn’t allow me to bend and threw off my body proportion. My knee and hip contractures wouldn’t allow me to straighten out. Then there’s the fact my bones are crap and could never withstand weight. I would still probably seek the cure/treatment though because if I could get enough back to be totally self sufficient I would be beyond thrilled.

    When Spinraza came out, even though I tried keeping my expectations low the “what if’s” crept in. I’ve learned I have to be good where I’m at and anything else is a bonus. It’s kind of like when the lottery jackpots get up there. It can be kind of fun imagining winning, but I don’t bank on it.

    • brianna-albers

      Member
      June 7, 2019 at 3:02 pm

      I really love this perspective. I feel the same – cures are fun to think about, but the reality of being “cured” (whatever that ends up looking like) is, in my opinion, way more complicated and emotionally loaded than we make it out to be.

Log in to reply.