AVXS-101 (Zolgensma) Trial on Hold
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?