Why Participating in Research Studies Is Important to Me

For as long as I can remember, I have participated in the SMA community in some way. Whether it was opening a lemonade stand as a little girl, participating in a research study, or running a nonprofit organization as an adult, I have always been committed to making a difference in advancing lifesaving treatments for my disease.

From raising money to giving my time, energy, and sometimes even blood to research labs, my intention has remained the same: I want to lend more hope to the future of SMA.

When I set out to participate in research studies many years ago, it was to help pave the way for those who followed in my footsteps. These studies were a large part of my upbringing. It was a time when treatments didn’t exist and hope was nothing more than a “someday” feeling.

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Given the unique nature of my SMA, I never expected to witness or experience a treatment in my lifetime. Every time a clinical trial became available, I never met the criteria. Although I was diagnosed with SMA type 1, I didn’t quite fit the benchmarks of the diagnosis. It was disheartening, to say the least.

Nevertheless, I still showed up and vowed to participate in whatever research study was available. If not me, then someone my age would someday benefit from the work being done.

Despite never knowing the outcome of my participation, I persisted with these studies. I did it so that someday, parents wouldn’t receive a grim diagnosis for their child like my parents did. I did it for their child and so many others who now have access to therapies and treatments early on in life.

Deep down, I longed for a treatment for myself. But I had accepted the fact that this would likely not be possible in my lifetime.

Then, in 2016, my life changed forever when I heard the news that I was eligible to receive Spinraza (nusinersen). I have since received 17 doses of this treatment, but in the years leading up to that defining moment, I felt as though I’d never have my chance.

Now, almost five years and many lumbar punctures later, I hold on to that feeling, for I know others are still waiting for their chance. Knowing this motivates me to continue volunteering for research studies, even if my participation plays the smallest role. After all, the SMA community has taught me that we’re all in this together.

And it’s together that we will create change.

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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, and are intended to spark discussion about issues pertaining to spinal muscular atrophy.