Not Everyone Who Has SMA Wants Spinraza … and That’s OK

Kevin Schaefer avatar

by Kevin Schaefer |

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Kevin Embracing my inner alien
December 23, 2016 was a big day for my family and me. Not only did my sister go into labor with she and her husband’s first child on this day, but it also was the day it was announced that the first FDA-approved treatment for spinal muscular atrophy was made available to the public. Since then, this treatment, called Spinraza, has been a prime point of discussion within the SMA community.

Some of you may be baffled by the title of this column. You may be thinking, “Why in the world would someone who has a severe neuromuscular disease not jump at the chance for some sort of treatment?” I understand the confusion some of you may feel toward this notion, but it’s important to look at this from a multifaceted perspective. Furthermore, people need to realize that not every person who has SMA is the same. Each of us possess a unique set of personalities, goals, desires and perspectives.

For the past few months, I’ve had multiple conversations with other SMA adults regarding Spinraza. Some were elated by the news back in December, and have either started to undergo treatments or are working on getting it approved by their insurance providers. Others, however, are either skeptical about Spinraza, or simply are uninterested in seeking the treatment for a variety of reasons. While all of us are overjoyed by how many kids and newly diagnosed patients already have received Spinraza and have yielded tremendous results, those of us who are SMA “veterans” have many things to consider before going through the treatment process.

It’s complicated

Having lived with SMA my entire life, I can tell you that I have a complicated relationship with my disease. When I was first diagnosed at 18 months, it was a nightmare for my parents. Not only did the doctors tell them I would never walk and that my muscles would decay over time, but they also were told I had a maximum life expectancy of 18 years. This is typically how most SMA stories begin, and it would cause any parent to shutter.

Over time though, the possibility of a “normal” life became a reality for myself and many others. I went to school, made plenty of friends, got involved in different things and drove my parents crazy as a teenager. Now, as a 23 year-old college graduate working on a writing career, that initial stage of terror and hopelessness my parents experienced when I was first diagnosed seems more like a faint memory. I know how to handle SMA on a daily basis, and there are very few obstacles I face that come as a surprise or that I don’t know how to deal with.

It’s because of this that many SMA adults have a bittersweet reaction to the notion of a treatment. On the one hand it’s a blessing for younger and future generations of SMA patients and their families. Most families I’ve talked to who have young or newly diagnosed patients have been able to get their children the Spinraza treatment without any insurance battles, or other bureaucratic obstacles. On the adult side of the spectrum, many of us (including myself) have been denied by our insurance providers on the basis that we’re too old to receive the treatment. Others SMA patients simply don’t want to go through it at all, as it is a matter of both time and cost that would interfere with their work and personal lives.

OK with waiting

I fall somewhere in between wanting Spinraza and not wanting to pursue it at the moment. While I’ve been denied by insurance, I’m also OK waiting a little longer to receive it or some other treatment down the road. Right now I’m more focused on my career, getting a new wheelchair and hiring more caregivers for the future. As I mentioned earlier, receiving Spinraza is a costly and time-consuming process, which for some SMA adults is just as burdensome and draining as the actual disease.

I’m in no way trying to say that SMA isn’t as brutal and frustrating for adults as it is for those who are newly diagnosed. There are days when it takes a toll on me physically, emotionally and mentally. There are days when all I wish for is the ability to raise my arms on my own and hold someone who I care about. Just because I’ve lived with this disease my whole life and know how it works, doesn’t make living with it any easier.

All I’m saying is that the introduction of a treatment is more complicated than most people realize. I am certainly glad that Spinraza exists, and that we’ve already seen incredibly positive results from it across the SMA community. Yet, at the same time, I want people to understand that it’s OK for people who have SMA not to want the treatment.

We all may share the same neuromuscular condition, but it doesn’t define who we are. We’re still individuals and, treatment or no treatment, our decisions are our own.


Note: SMA News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of SMA News Today, or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to spinal muscular atrophy.


Ashley avatar


If you get denied twice by your insurance company, you can get onto Biogen's Free Drug Access program. They won't deny any patient. However you still need to pay for the prodecure iteself which varies for everyone but is about $4,200 for each procedure. That does add up but is well worth it in my opinion.

Pamela K. Muhammad avatar

Pamela K. Muhammad

Hi Kevin great and thorough explanation! I have mixed feeling as well about the possibility of spinzana treatment. Have a great day!

Kristin avatar


My thought is that I'm not doing this for a "normal" life. BUT I can't handle eventually losing the ability to talk, swallow, or breathe. Going through this I understand the time and money it takes up. I get waiting... but I don't see why giving up on the treatment [or future treatments for that matter] completely is something somebody would want to do. Biogen is constantly helping out financially and I can't see the positive in not eventually improving your health.

Claudia avatar


I have SMA and don't want the Spinraza. I'm 32 and after a long time period of study (PHD) I reconsidered the big unknown choice of a writer's life.
Right now I'm on the brick of hell, because I feel guilty about my choice to not get this treatment. Believe it or not, I didn't put the wheels on the floor of an hospital in 10 years, and this peace of mind + physio + avoiding seasonal illnesses, well... It's kinda make me just feel "happy" and even "stabilized" because even if I'm still dependent, I'm not loosing physical strengh.
But I think about my parents and I'm afraid to get to the point to struggle with them because I don't trust the side effects, the potential consequences and the lost of control on my mind, body and life.
I recognize myself in your article and I thank you so much for sharing it. I understand why you have chosen the profession of writing : you need, as I need, to express what's beyond. Expression is what is left, and like you, I'm done with silence.


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