I always hoped and wished for a day that we would see incredible achievements in the medical world for the SMA community. I never knew if I would be alive the day we found a cure or even a treatment.
Back when I was first diagnosed over 30 years ago, they hadn’t even discovered the gene yet. We were reliant on muscle biopsies to diagnose with very little information since this was a new disease to our family: spinal muscular atrophy.
Finding treatment options for SMA
As time has gone by, many people have devoted their time and efforts to finding treatments for this disease. I remember when I first read an article about a possible treatment for individuals with SMA.
I remember in the beginning stages of the treatment--there being talks of “this is only for children,” or “this will only work if you catch it early.” And although I am a firm believer in newborn screening to help treat infants as soon as possible, that didn’t feel like a whole lot of hope for me as a woman living in her 20s with SMA.
I began to do some more independent research and contacted the companies distributing this incredible new treatment. I then found that they too were excited to see adults with SMA could potentially use this new treatment.
Advocating for myself to try new treatment
I remember when I began the process and started applying for this new and incredible treatment. Many of my doctors were unaware of the treatments; this was NEW.
In some cases, I was bringing the treatment option to my doctors and having them write the script for it. I remember that we finished an application, submitted it, and I didn’t think twice about the approval of it. I had the mindset of, “Well, why wouldn’t they approve it? This is a life-changing drug.”
Insurance denials and appeals
I also remember the day that I received a denial. It felt like there was no hope in store for me to rest. I received three denials before we went a new route. I remember appealing to every single one of them.
I was not going to quit. This multi-million dollar drug seemed as though it was out of reach.
Full of hope when approved for treatment
But then one day I received another phone call. A call where a judge, a doctor, and a representative for the treatment sat on the phone and fought for me; they explained to the judge why they believed this treatment was going to be beneficial for me.
I remember getting chills, hearing the words at the end: “By the power vested in me,” (I felt like I was getting remarried), I would like to approve you for this drug.” It was one of the happiest days of my life, full of hope. I felt hope again.
No matter what this journey looks like for you, know that it will all be okay in the end, despite the ups and downs.
Have you found an SMA specialist you trust?