Early Identification Cuts Costs for Spinal Muscular Atrophy

TUESDAY, June 14, 2022 — Early identification and treatment of patients with spinal muscular atrophy can reduce the total associated financial costs, according to a study published online June 8 in Developmental Medicine & Child Neurology.

Tamara Dangouloff, from the University of Liege in Belgium, and colleagues compared differences in the societal financial costs and quality of life between untreated patients with spinal muscular atrophy (93 patients) and treated patients identified because they presented with symptoms (42 patients) or were identified by early testing (sibling or newborn screening; 14 patients).

The researchers found that total costs were lower in untreated patients due to the high cost of drugs used in treated patients. Treated patients who were identified by early testing had lower costs than treated patients identified because they presented with symptoms. Patients with two SMN2 copies had higher costs than those with more copies across all groups.

“These data are important as they are issued from a real-life prospective collection,” a author said in a statement. “They demonstrate clearly that as long as the decision to reimburse treatments for spinal muscular atrophy has been made, newborn screening becomes a no-brainer — not only because it gives patients a much better future, but also because it saves a significant amount of money for the taxpayer.”

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