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Spinraza (Nusinersen) Also Effective for Later-Onset SMA
Hey everyone!
I came across this article earlier, which examines the evidence that Spinraza (Nusinersen) is effective in later-onset SMA. Seeing this data is really encouraging to me, despite the age range looked at still being young (in my opinion,) 2-15 years old. What do you all think?
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3 Replies
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That would have been the age range when I first started showing symptoms. I always had to plan my future where I knew I’d be losing abilities, but maybe these kids won’t have to experience that.
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One other thing about these results. They’re using the HFMSE to evaluate type 3s. If it’s the same test that they have me do during my 6 month evaluations, it doesn’t really work well for type 3. Even before I started Spinraza I was able to complete about 80% of the tasks, with my stronger arm able to do everything they asked. It doesn’t leave much room for showing improvement when you’re already at the maximum so I wonder if that’s why the change showed for type 3s was lower than type 2.
My left arm was has always been able to lift about 2-3 pounds, but the maximum in the tests I did was 500g or about 1.1 pounds, and even then it wasn’t lifting the weight, but sliding it, which I could do easily. So if my left arm is improving, there’s no way to determine that using this method which of course makes it looks like nothing is changing when the results are reported.
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Very valid point Lupa. I know as a type 2 when I was able to show improvement the gap between where I’m at and where I need to be for it to actually count is so much that the little improvements don’t score. I think it’s really difficult to find a standardized test that works for everyone.
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