Friday, August 11, 2017

What is Spinraza, anyway? (and why is Nate even in a wheelchair??)

We took Nate to Children's Hospital in Milwaukee on August 9th for his first treatment of Spinraza. This is the first and only FDA-approved treatment for SMA, which is a genetic disease in which a lack of survival motor neuron protein affects the part of the nervous system that controls motor function.

That is why he uses a wheelchair. SMA stands for Spinal Muscular Atrophy, and Nate has SMA 3. 

So this wasn't our first trip to Children' took a long time and lots of testing to get a diagnosis. This all happened a few years ago, and up until December of last year, there was no treatment for SMA. Basically,  the lack of the SMN1 gene leads to deterioration of nerve endings and his muscles will eventually atrophy. 
That's why his legs are not strong enough to walk, or even bear weight. 

We have been waiting a long time for Spinraza to be approved! 
Then after we learned it was finally approved, we had to have him tested again for his insurance to review. At the time of his testing, his insurance didn't even have a policy in place yet for Spinraza. 

More waiting!

But we finally got the call the Nate was approved! This happened in early July, which led us here, to this first appointment! 
It was an all-day affair; blood tests, long meetings with his neurologist,  more waiting... we played hide and seek in the garden....
...took naps in the sun....
and then finally we headed to radiology, where he was so brave and good...I was really proud of him.

Natie! I'm like, let me take a picture to remember this historical day!
But truly, we feel honored to be one of the first for sure in Wisconsin to have access to Spinraza, and one of the few in the country already beginning this treatment. 

We go back in 2 weeks for another treatment...2 weeks after that, 4 weeks after that, then every 4 months thereafter.
If it works, it will stop the progressive loss of muscle tissue, and what he is able to do he will be able to maintain. In other words , he will not continue getting weaker.

The drug is administered via spinal tap, so he is sedated first. Awww....little man!
Past that, we don't know much more. The treatment is so new, and the drug trials that were performed before it was approved were all done on infants with SMA 1.

None of the trials kids had Ds, either. Nate is making history and his charts and progress will go down in medical records for future Spinraza studies!
We are SO GRATEFUL for all of your good wishes and prayers! It has been awhile since I posted anything on this blog, but I'll be sure to keep it up-to-date with future treatment info and progress reports!