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What is something that you hope to see in the next ten years with SMA treatments?
I’m just sitting here thinking about how much the landscape for SMA has changed in the last ten years alone. We’ve had several treatments approved, breakthroughs in medicine, and amazing outcomes in the community.
It is truly mind-blowing in the best way, and it really feels like the trajectory of this disease is changing right before our eyes. If that’s all that we can accomplish in ten years, imagine what else is possible, especially now with AI at the forefront.
What is something that you hope to see in the next ten years with SMA treatments?
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4 Replies
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I think the next most important step in SMA treatments will be motor neuron regeneration or creation. Not a single treatment developed up to this point, and I celebrate and appreciate every one of them, can undo prior damage. That means they will have limited efficacy. Even Ivitsma, which I am not personally pursuing, will most likely stop progression (assuming the patient doesn’t develop antibodies), which is great, but can’t correct or undo prior degeneration.
We are fortunate to have treatments and options, but this next important step is my hope, which was the question posed.
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Agreed. I was thinking the same thing.
I know this isn’t specifically related to treatments, but I hope that in the future we see more established care for SMA with adults. Now with treatments, children with SMA are becoming adults with SMA. Life expectancy is increasing, and people are thriving, but I’m finding that there aren’t many programs in place where doctors have experience in adult SMA care. It’s all so new. It’s a great problem to have in the respect that we have these treatments available to us. Truly a blessing. But it’s something that needs to be addressed nonetheless.
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agree. motor neuron regeneration is a priority. i think in 5 to 10 years it will reach human trials as a priority for adults and sma europe asked for proposals about this topic
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Yes, I can definitely see this happening too, especially now that AI is available to us. I think more breakthroughs are on the horizon.
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