Being alive to witness history in SMA research, treatment

When I was born 31 years ago, spinal muscular atrophy (SMA) was a very different disease than it is today.

There were no approved treatments. No gene therapy. No medication designed to slow its progression. For those of us born before the era of SMA treatments, the reality was painfully simple: We learned to do what our bodies allowed us to do, knowing SMA could eventually take some of those abilities away.

The disease was once considered the leading genetic cause of infant death. For families receiving an SMA diagnosis decades ago, the conversation could be devastating. Instead of being given treatment options and hope for the future, parents were given a life expectancy.

Imagine being handed your newborn child and being told how long they might live. Imagine bringing your baby home knowing medicine had little to offer. That was the reality for far too many families.

Today, that conversation can look dramatically different.

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Changing the trajectory of SMA

When an SMA diagnosis is confirmed, treatment is now one of the first things discussed. Families can learn about available therapies and how early treatment may help preserve motor function. Instead of being told only what SMA might take away, parents are told what can potentially be done to change the course of the disease.

Science kept moving. Researchers kept asking questions. Families kept advocating. Patients participated in clinical trials and shared their experiences. Scientists continued studying the genetics and biology behind SMA. Decades of research eventually turned into something that once seemed almost unimaginable: treatments.

In 2016, the U.S. Food and Drug Administration (FDA) approved Spinraza (nusinersen), the first SMA treatment. In 2019, Zolgensma (onasemnogene abeparvovec-xioi) became the first FDA-approved gene therapy for SMA. In 2020, Evrysdi (risdiplam) became the first oral treatment for SMA. Then, in 2025, the FDA approved Itvisma (onasemnogene abeparvovec-brve), expanding gene therapy to pediatric and adult patients ages 2 and older.

I was born into a world where there was no treatment for SMA. I have lived long enough to watch scientists go from trying to understand this disease to developing therapies that can change its trajectory.

That is more than scientific progress. That is history. And I feel incredibly fortunate to be part of a generation that has watched that history unfold.

We remember when SMA meant progression with very few options. We remember the uncertainty. We remember watching abilities disappear. We remember when treating the underlying cause of SMA seemed like something that belonged in the distant future.

Children diagnosed today may receive treatment before significant motor neuron loss occurs. They may grow up never knowing the same version of SMA that previous generations knew. They may have opportunities that people like me could only have imagined.

That thought is incredibly emotional for me because when I think about the children coming after us, I don’t want them to understand SMA primarily through loss. I want them to know SMA as a treatable condition. I want them to have the opportunity to discover what their bodies can do without automatically assuming that every ability comes with an expiration date.

Of course, treatment is not the same as a cure. SMA still exists, and people living with it still face challenges and uncertainty. More research is needed.

But that doesn’t diminish how extraordinary this moment is. If anything, it makes me appreciate it even more.

Witnessing history

I often wonder what my younger self would say if she could see where SMA research is today. Would she believe there would one day be multiple treatments? That gene therapy would become a reality? That children with SMA could have opportunities that previous generations never had?

I think she would be amazed.

Maybe that is the greatest gift of being part of this generation: We don’t just get to hope for the future. We have watched the future become reality and witnessed history.

And while I wish I could have been born into a world where SMA treatments already existed, I am grateful that I have lived long enough to see the world change. Because perhaps one day, another generation will look back at ours and wonder how we ever lived without a cure.

I hope they do. And I hope they understand that progress didn’t happen overnight. It happened because people kept researching, advocating, asking questions, participating, and believing that the future could be different.

I am living proof that it can be.

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.