Advances in SMA treatment are like something out of science fiction

Did you know dogs can be diagnosed with SMA?

I did. As a child, I used to stare up at a news clipping of research about the condition in canines, which was pinned to the wall in the office of the Muscular Dystrophy Association (Singapore) (MDAS). My recollection of its exact contents are mostly fuzzy — and I haven’t been to the office in years — but I do distinctly recall the article saying that some scientists hoped what they learned about the canine version of SMA would also help to treat humans.

It’s wild how far we’ve come in just over a decade. I’m 26 now. While dogs sadly don’t have any disease-modifying therapies (DMTs), humans have three and counting. It sounds like science fiction, but it’s not.

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Cyborgs in the flesh

As I sat in the gorgeously golden banquet hall at the Marina Bay Sands hotel for MDAS’ 25th anniversary gala last week, I couldn’t help but think of that news clip and the difference that one or two decades can make. My kid self hoped we could one day treat SMA directly, in whatever species, but I didn’t believe my adult self would live to see it happen. I wondered similarly if the founders of MDAS — which was originally headquartered at director Sherena Loh’s home, before it ever had an office — could’ve imagined the organization would grow so far past its small and humble origins.

It’s not like neuromuscular disabilities are as common as the flu. Recognition and research funding for each specific disease under this umbrella is therefore always hard-won. Otherwise, these diseases perhaps would be close to eradicated by now. But that doesn’t mean we aren’t on our way there, or that we know how far DMTs and other supplementary treatments can carry rare disease patients.

At the MDAS gala, leaders from the organization marked its 25th birthday with the official launch of their Cybernics Treatment program. Upon hearing that the therapy utilizes a Hybrid Assistive Limb (HAL), I envisioned something like the assistive JACO robotic arm that my American SMA peers have tried. I thought the user would be wielding a high-tech appendage like a puppet, or vice versa.

But the three-minute demo of the Cybernics Treatment program stunned me. Developed by Cyberdyne, a Japanese robotics and technology company, HAL is actually a device that attaches sensors to its user, allowing it to read the user’s intended movements via bioelectrical signals. It then assists in joint movements, granting the user more range and “strength.”

In essence, HAL is a workout coach and buddy in the program — which allows neuromuscular patients to exercise independently by temporarily transforming them into cyborgs.

“Imagine how much independence SMA patients could regain,” I thought, “especially in combination with DMTs like Evrysdi (risdiplam) and muscle-directed drugs like apitegromab.” The frustration I felt earlier in the evening at my inability to do my makeup on my own could be eliminated in a few years.

I nodded along as the gala’s emcees talked about the “next chapter” in treating rare neuromuscular diseases.

Minds over matter?

As thrilling as this next chapter is, I admittedly also find it terrifying. Science fiction stories don’t always end well. Quite the opposite, if you look at the entire subgenre of dystopian fiction.

I worry that patients, caregivers, and healthcare providers will pin far too many hopes on the ever-evolving technology we have — or that, sometimes, we might not hope enough to keep pushing ahead. I worry we may end up framing neuromuscular diseases as aberrant when discussing how the technology helps us, and how that might affect patients’ mental health. And most of all, I worry about gaps forming in the SMA community as some patients react better or have more access to newer therapies than others.

We would do well to remember we must prepare ourselves mentally and emotionally for an unexpected future. The arc of progress is speeding along with or without us. But just maybe we’ll end up with more good than bad in another 25 years.

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.