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Despite Taking Spinraza, SMA Type 1 Children Show Nonmotor Problems
Despite its well-reported benefits in motor function, Spinraza (nusinersen) may not be as effective at preventing or easing nonmotor complications, such as facial deformities, feeding problems, and scoliosis, in children with spinal muscular atrophy (SMA) type 1. Given that all these complications may affect the child’s lung function and health, they should be closely monitored to avoid further respiratory complications — the main cause of death in this patient population — the researchers noted.
These were the findings of a small Israeli real-life study, “Nonrespiratory complications of nusinersen-treated spinal muscular atrophy type 1 patients,” published in the journal Pediatric Pulmonology.
SMA type 1 — a severe form of the disease with an onset before 6 months of age — is characterized by progressive muscle weakness and wasting that affect not only a person’s motor function, but also their ability to breathe and eat.
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