Differences in swallowing efficacy of disease modifying treatment between infants receiving pre-symptomatic and symptomatic administration

Background: Spinal muscular atrophy causes progressive motor neuron degeneration that impedes an infant's ability to maintain full oral nutrition and manage secretions. Development of pharmaceuticals that halt neuromuscular degeneration have enabled survival and improvement in motor function, with infants who receive treatment before symptoms exhibiting better outcomes than those who receive treatment after symptom onset. Little is known about the impact of treatment timing on swallowing. We retrospectively evaluated swallowing biomechanics and function among infants who received a disease modifying treatment and a swallow study as part of routine clinical care at 13 international children's hospitals.