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"Rapid Motor Function Improvement" article
So in the article about Zolgensma results that was posted today, it starts off with
Treatment of presymptomatic babies with the investigational gene therapy Zolgensma provides rapid improvement in motor function in infants with spinal muscular atrophy (SMA) types 1, 2 and <u>3</u>, according to preliminary results of a Phase 3 trial.
Infants do not have SMA type 3 by definition. I think the author thinks SMN2 copies are what determines the type and that’s not true. The article itself is fine after that since it mostly just copies the press release from the company which mentions only type 1.
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3 Replies
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Lupa,
I can understand your confusion. As of right now, the only thing that I know that they are allowed to say regarding ZOLGENSMA, and by them, I’m speaking about AveXis, is that only infants were the ones there were in the preclinical trials. With regards to Type 2 and Type 3 SMA, not really a lot of information has been given. And to be honest with you, until ZOLGENSMA gets approved by the FDA, AveXis will probably not give out any information regarding the effects of this potential treatment outside of their initial Type 1 scope.
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It wasn’t confusion. The author is wrong saying it was tested on types 2 and 3. It wasn’t. It’s the same trials they’ve been doing on type 1. So the author mentioning types 2 and 3 could confuse other people into thinking they’d expanded the trial.
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I think typing by age of onset and milestones reached will have to go by the wayside. With newborn screening becoming more prevalent and treatment options becoming more widely available it just won’t work the same. It’ll be interesting to see how pinpointing type evolves.
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