Page 16 - Mobility Management, November/December 2021

ATP Series
The last time Cure SMA — a national organization that supports people with spinal muscular atrophy (SMA) and their families — held its annual conference in person was June 2019.
Spirits were high at that event in Anaheim. For the first time, there were two approved treatments for SMA, which is the most common genetic cause of death in infants worldwide. Spinraza (nusinersen), the first SMA drug therapy and one that requires ongoing administration, had been joined by Zolgensma, a one-time gene therapy treatment administered by intravenous infusion.
Kenneth Hobby, Cure SMA’s Executive Director, spoke of new horizons for the adults (Spinraza) and children (Spinraza and Zolgensma) taking the treatments.
“It’s not going to be the SMA we’ve known in the past,” Hobby told conference attendees. “In some ways, SMA is going to be new. New issues are going to be created.” He suggested that SMA symptoms might get milder as a result of the treatments, but that some issues — such as significant scoliosis — would likely remain.
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The Pandemic’s Effect
The pandemic that has prevented in-person conferences for Cure SMA the last two years also made it more difficult for some SMA patients taking Spinraza to access those treatments on time. In addition, pandemic lockdowns made it more difficult for some patients to regularly receive physical therapy.
A September 2021 study published in Frontiers in Neurology examined the functional impact when children’s nusinersen doses were delayed (after four initial loading doses, nusinersen is administered three times per year). The study — Effect of the COVID-19 Pandemic on Children with SMA Receiving Nusinersen: What Is Missed and What Is Gained? — was led by Italian researchers who studied data on 25 children, aged 2 to 15, who were on a Spinraza regimen when the pandemic began.
Marisa Wexler, MS, writing for SMA News Today, said eight
of the children experienced delayed Spinraza infusions due to pandemic lockdowns, with those postponements ranging from 26 to 91 days. Researchers “noted that there was no clear associ- ation between these treatment delays and changes in functional
SMA:
WHAT’S
NEXT?
How Treatments for Spinal Muscular Atrophy Could Impact Mobility
By Laurie Watanabe
MobilityMgmt.com
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