Trofinetide Use and Treatment Patterns Among Children Aged 2-4 Years with Rett Syndrome in the United States: A Retrospective Specialty Pharmacy Linked Claims Database Analysis

CONCLUSION: In routine US practice, approximately two-thirds of children aged 2-4 years initiating TROF remained persistent during available follow-up, with sustained use beyond 1 year and a subset (14.5% of non-persistent children) restarting after discontinuation. Non-persistent children had significantly higher baseline rates of dysphagia, gastrostomy, and breathing irregularities, suggesting that early disease-related multisystem complications may be associated with reduced treatment...