Playing the Evrysdi Long Game

Growing up, it always seemed like there was never going to be a treatment for SMA aside from the normal physical therapy, respiratory medications, etc.

But nothing was specific for just us and at some point, I became okay with the idea that maybe there would never be anything else.

New medications and treatment options for SMA

Then, there was a sudden burst of medications being released such as Spinraza and Evrysdi (Risdiplam). At first, I’ll admit I was very hesitant.

I’d made it this far, why change or add something new? What exactly was it going to do to my body?

Unfortunately, I never participated in the Spinraza treatment as my spinal rods would make it difficult to find a port to administer the medication.

Also, one of the potential side effects is kidney issues. Since I only have one kidney, my doctors and I weren’t willing to take the risk.

The payoff, at least to me, just wasn’t big enough.

Waiting for a treatment option that could work for me

So, I waited some more with the rumor of Evrysdi on the horizon, a medication that’s taken by mouth at home. This seemed like a promising possibility.

As soon as Evrysdi was released, I was probably the first on the phone with my neurologist asking what needed to be done to start.

I won’t get into the insurance hassle (at least not for this article), but my first shipment arrived in that glorious, Styrofoam cooler a couple of weeks later.

How will this medication help me?

Each bottle literally looked like liquid gold to me. Over twenty years of waiting and there is finally a medication that’s supposed to do, well...something.

I didn’t know what that something was to me, an SMA adult with type 1-2. I have 3 SMN copies so I have a little more strength than a typical SMA type 1.

Either way, was I going to miraculously stand up from my wheelchair? Probably not.

But that question is almost not impossible to ask as the SMA community sees babies crawl, walk, and run around because of early intervention. Of course, not everyone is going to experience such positive gains.

Will I notice any changes from this treatment?

But I wanted something. Maybe my speech would get better. Maybe my swallowing would improve and I wouldn’t need to rely so much on my g-tube.

Maybe I’d see some improvement with my hand strength so I could have more stamina on the computer.

Honestly, one of the above seemed great. All the above would be a miracle.

During the first few months of treatment, I definitely noticed a change in my speech. My tongue didn’t get so tired or seize up when talking for long periods.

Also, at first, I did experience a change in eating with a slightly greater appetite and ability to finish a meal.

How I'm feeling a year later

But a year later, I mostly feel the same. Some days I have a lot of stamina and some days I don’t. Some days it’s easier to drive my wheelchair and others I need help just getting around the house.

It’s really frustrating because now that I have this medication I want all its capabilities. I want to feel the best I’ve ever felt.

Of course, I understand that any treatment at my age is just for maintenance, so I don’t lose more ability or strength.

So, when my doctors ask how I’m doing, I guess my answer is, “Eventually I’ll let you know.”

For now, I have to play the long game and hope it works.