SMA News Forums Forums Treatments and Research Zolgensma (onasemnogene abeparvovec) AVXS-101 (Zolgensma) Trial on Hold

  • AVXS-101 (Zolgensma) Trial on Hold

    Posted by deann-r on November 5, 2019 at 11:15 am

    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?

    alyssa-silva replied 4 years, 6 months ago 4 Members · 3 Replies
  • 3 Replies
  • robert-stump

    November 5, 2019 at 7:46 pm

    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.

  • halsey-blocher

    November 5, 2019 at 9:46 pm

    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.

  • alyssa-silva

    November 6, 2019 at 8:52 am

    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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