Last week was a very exciting one within the SMA community. The FDA approved Evrysdi (or risdiplam, the name used during development), the third ever treatment for SMA.
Before this past week, there were two existing treatments – Avexis and Spinraza. I’m not going to go into too much detail about either, because I’m not an expert and I don’t want to say something that’s actually wrong, but I can explain a little bit about each. Avexis is actually gene therapy, and it’s the closest thing to a “cure” that there is. It’s relatively new, incredibly expensive, and because it’s gene therapy, it can only be used on children under two years old.
Spinraza is able to be used by all ages, and all types of SMA (see my first post for more information on types), but it’s administered intrathecally, meaning a spinal injection. This is not easy for anyone, but is even more difficult for people with SMA, many of whom have had spinal fusion surgery as a teen to correct scoliosis. So while Spinraza is approved for adults, not all are able to easily get it because of the complication created by spinal rods. With Spinraza, there’s also no guarantee that you’ll gain any strength back – it’s intended to stop or slow progression. So the younger you start it, the better. There are some side effects, too – one of which is a spinal headache, which can be pretty debilitating while it lasts.
I had an additional complication in that my neurologist decided that she just… wasn’t going to dose adults with Spinraza, period. It was an incredibly frustrating decision, and one that I still don’t agree with. But when we heard that risdiplam was on the horizon, we decided to wait and see what would happen with that before we decided how to appeal her Spinraza decision.
So the news on Friday was incredibly welcome. Like Spinraza, Evrysdi isn’t a cure, and while some people do see improvements, the goal is to slow or stop progression. Unlike Spinraza, though, it’s not given through a spinal injection – it’s an oral, liquid medication that you can take at home. Not only is it more convenient, but it makes it so much more likely that I’ll be able to get it! And it’s been approved for anyone over two months old.
I know that there’s no guarantee that I’ll have any improvements from Evrysdi, but honestly, knowing that I won’t get worse is hugely important to me. SMA is progressive, but it happens in fits and starts, so I don’t necessarily notice that I’ve lost strength until months or years later. And there’s no way of knowing when it’s going to happen, either. So having the peace of mind to not have to worry about what strength I’m going to lose next year, in five years, in 10 years… that’s really important to me. I’ve come to accept that I’ll never be fully independent, but that doesn’t mean that I don’t want to hold on fiercely to shreds of independence where I can. Evrysdi would help me do that!
I’m sure it’ll be weeks (or even months) before I know what the next steps are. But I’m feeling hopeful, and that’s a really big thing right now.