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    Adapting to Life Outside Our Home With SMA

    I once heard a joke that a woman’s mind is like an Internet browser that has 1000 tabs open and running at once. I laughed at the time, but came to realize that if I didn’t actually feel like this before hearing the short acronym “SMA,” I most certainly do now.

    I spend much of my time pre-planning and thinking of possible problems that we may run into because of the kids’ diagnoses. I think many other SMA parents do this as well. Prior to our lives with SMA, we didn’t realize just how inaccessible the world is to those who are differently-abled.

  • This topic has 3 replies, 4 voices, and was last updated 2 months, 2 weeks ago by Alyssa Silva.

    • Author
      • #21197
        DeAnn R

        I’m sure you’ve heard by now that one of the trials for AVXS-101 is on hold.  You can read more about it here. From my interpretation there are negative side effects from the IT delivery method of the drug commonly known as Zolgensma in animal studies. IT is the delivery method they’re looking at for those of us older than 2 years old. On one hand it’s frustrating. It seems so close but now it’s slipping through our fingers. On the other hand I’m glad they’re taking the time to get it right. Besides that, there are other drugs in the pipeline. Zolgensma however is the only one that I’m aware of that targets the root cause of SMA. I know I’ll be keeping a close eye on further developments. What do you think about the news? Would it make you hesitant to participate in future human trials?

      • #21226
        Robert Stump

        It makes me sad to learn that Zolgensma may not ultimately be viable for us adults, and yes it definitely slows me down when considering partaking in any future trials for this drug.

      • #21231
        Halsey Blocher

        This is a bummer. I’m already consider Risdiplam as better option for me, but it’s still disappointing. However, I have to agree that it is best that they are finding these problems now before it’s used on people.

      • #21236
        Alyssa Silva

        That is definitely disappointing to some degree, especially since this drug holds such promise in essentially correcting SMA. However, I’m not sure it would deter me from taking it down the road just because, as Halsey said, they’re finding all the problems now as to prevent them from happening in adults.

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