[andrew]

For me when I was on Spinraza I always felt a boost in stamina/energy almost immediately after the LP and for the first few days even though there was a recovery period for my lower back that ranged from a few days to a few weeks I always felt the effects of the medicine the most in the first few days. I feel like it still worked pretty well for me the first few weeks but it felt like a slow decline and around the 45-60day periods most of the effects always felt very noticeably diminished. And then waiting until the next LP the last few months was always a struggle.

I used to be on Spinraza for I think ~ 3years before I switched to Evrysdi (Risdiplam) around March of last year. I’ve personally been a big fan of Evrysdi since I’ve experience many similar benefits from both medications with some more additional noticeable benefits for swallowing both solids/liquids from Evrysdi. The daily consistency on Evrysdi has felt really nice. And I also haven’t had any downsides on Evrysdi (I see a lot of people experience some GI issues but I didn’t get any from Evrysdi – I have a lot of GI and motility issues normally and Evrysdi didn’t aggravate them for me, if anything they maybe feel slightly better on Evrysdi).

That’s been my short story of the medications I’ve been on. Let me know if I can provide any more info on anything relating to them. I sort of sidetracked into my Evrysdi experience even though the post asks about Spinraza x.x – but I figured it wouldn’t hurt to include.

 

[andrew]

I agree that the old SMA type system is super outdated and for me personally and has caused me issues. I was diagnosed with SMA type III when I was a child and the few years leading up to my growth spurt I struggled to walk independently and around the time of my growth spurt at age 12 is when I pretty much lost my ability to walk independently. Because I had the classification of type III a lot of my physical issues and inabilities were not addressed or helped with as much as they could have been (One big example is the fact that I was never physically able to use a public restroom since late elementary school but I never got help with that until after I turned 18 and qualified for the MA PCA program).

 

In short the old type system provides almost no benefit in terms of predicting future outcomes for disease progression and in cases such as my own actually cause a lot of my issues as a child to be overlooked. The SMN2 copy number system I do think does a better job of predicting disease progression/severity then the old one which purely looks a phenotypical snapshot of functionality at childhood. But at the same time like you said the SMN2 copy system isn’t completely reliable either and has a lot of things that are less well understood that can still impact disease severity and progression. And it’s 100% true that thinking of SMA as a spectrum of severity that changes over time is the most accurate way to look at it but currently medicine can’t accurately track something like that so they can’t put that into practice.

 

I agree that relying too much on any type of type system will probably lead to bad results. And every case of SMA should be monitored closely and on a case by case basis to address the physical needs of every individual. It’s always good if medicine continues to find more and more accurate markers that can predict disease severity/progression in order to plan for the needs of individual with SMA, especially in the case of youths with SMA who don’t have the same ability to communicate or plan for their future needs like adults can.