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.
Is self advocacy a part of your daily life?