2026 Public Health Laboratory Newsroom
The Breakthrough Saving Babies’ Lives: SMA Treatment

Spinal muscular atrophy (SMA) is a deadly genetic condition that affects an average of 6 newborns in Minnesota every year. The most common and severe type of SMA quickly destroys motor nerves, leaving babies unable to move, swallow, and eventually in many cases, breathe. These nerves cannot be repaired; any treatment must begin before most symptoms occur. 

Before 2017, the most common type of SMA, Type 1, led to 50% of afflicted newborns dying within 6 months and almost none surviving to age two. In 2019, a new combination of medications and gene transfer therapy revolutionized treatment for SMA. Now all Minnesotan babies with Type 1 SMA who get early treatment survive the first year. The more than 40 who have done so in Minnesota are all still thriving at this writing.  

The process of identifying SMA in newborns begins with the Minnesota Newborn Screening Program. Soon after SMA became treatable, it was added to the Minnesota Newborn Screening Panel. This is the list of conditions that the Minnesota Newborn Screening Program routinely screens for on behalf of nearly every baby born in the state. By analyzing just a few drops of blood, the program’s scientists can discover indications that a baby may have SMA or over 50 other conditions.

A screening test does not provide a diagnosis, however. After a newborn’s blood spot tests positive for indications of SMA, the Minnesota Newborn Screening Program contacts the baby’s health care providers and informs them about the recommended next steps. First, they must order more testing to confirm whether the newborn has SMA. If a diagnosis is confirmed, the baby must quickly be referred to a neurology specialist for treatment.

One such specialist is Dr. Randal Richardson of Gillette Children’s Specialty Healthcare in St. Paul, one of three SMA treatment centers in Minnesota, along with the University of Minnesota and the Mayo Clinic. Since SMA treatment became available, Dr. Richardson has helped many children with SMA survive and thrive. In a presentation to the Minnesota Newborn Screening Program, he discussed both the science behind SMA treatment and shared grateful feedback he has received from parents of affected children. Here are just a few examples:

“We are incredibly grateful for the newborn screening test. Because of it, [name redacted] was diagnosed and treated before the disease progressed too far. Without the screening, we likely would not have known until the condition had advanced to a point where irreversible damage had already occurred.”

“We are incredibly thankful for the Minnesota Newborn Screening crew without them we would have never known our son had SMA and missed a very important window. Our primary care provider would have had no idea how to proceed as we are her first SMA patient and they helped give her names and to get us to Dr. Richardson! Now we are a year and a half later and [name redacted] is running and playing as strong as can be thanks to Newborn Screening, Gillette, Dr. Richardson and our primary care provider.”

“I've always loved living in Minnesota. But I have never been so grateful than when our son, was born here and promptly diagnosed with SMA within mere days. Thankful to have a Department of Heath that cares about, is thoughtful of, advocates for (I could go on and on) the children and parents that reside here. Because of your hard work at what you probably consider is "just your job", the biggest and scariest phone call we've ever received turned into the best thing our son could receive: a life altering medication and a tenacious team of medical professionals. The time from Newborn Screening to the call to treatment was “Incredibles” quick. (For real, Dash from “The Incredibles” fast ...) So thank you. From the bottom of our hearts, thank you. Though it may be your job, for those of us impacted, it is life itself. Or a chance at a ‘normal’ life. We hope you have a wonderful holiday season, from our family to yours!”

Dr. Richardson also emphasized that SMA treatments are still being improved. Researchers are working to minimize side effects and enable even earlier diagnoses. While they can always get better, the new treatments for babies with SMA have proven a tremendous success story.

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