Arthur Beisang, MD, highlights trofinetide’s impact on Rett syndrome

Rett syndrome is a severe neurodevelopmental disorder that can profoundly impact a child’s quality of life, affecting nearly every system in the body. According to Arthur Beisang, MD, complex care pediatrician at Gillette Children’s Specialty Healthcare, patients with the condition face a wide array of debilitating symptoms.

These symptoms include diminished intellectual capability, loss of mobility, impaired communication skills, and, frequently, the onset of severe seizure disorders. The severity and manifestation of these symptoms can vary significantly from patient to patient, making the management of Rett syndrome a unique and ongoing challenge for both clinicians and families.

A major breakthrough in addressing these challenges has been the introduction of trofinetide (Daybue; Acadia Pharmaceuticals Inc), the first medication specifically approved for Rett syndrome. Before its availability, treatment primarily focused on symptom management and supportive care.

Clinicians and caregivers now have a targeted therapy aimed at directly improving a patient’s core functional abilities. The safety and efficacy of trofinetide were largely established by the pivotal LAVENDER study, and its clinical benefits have been continuously supported by over 2.5 years of daily, real-world use.

Beisang notes that patients taking trofinetide frequently demonstrate noticeable improvements in focus and attention, nonverbal communication, and both fine and gross motor skills. Because Rett syndrome has such a highly variable presentation, the medication’s effects differ for every patient: Some experience significant developmental benefits, while others may see smaller yet meaningful gains.

When monitoring patients on trofinetide, clinicians historically had to remain vigilant regarding adverse effects. The traditional liquid formulation carries a notable risk of diarrhea, requiring close management of gastrointestinal distress and weight loss. However, a new, powdered stick formulation has recently entered the market.

By removing certain ingredients known to cause stomach issues, this new formulation has shown encouraging early results with a significantly reduced incidence of diarrhea. Furthermore, the new powdered Daybue Stix offers much greater flexibility and convenience.

Unlike the traditional liquid medication, the powder does not require refrigeration, vastly easing the logistical burden on caregivers and integrating more seamlessly into a family’s daily routine. As the first targeted treatment for this condition, trofinetide—and its evolving, patient-friendly formulations—represents a vital step forward in improving the everyday quality of life for children living with Rett syndrome.

Disclosure: Acadia Pharmaceuticals Inc.

Reference

Ebert M. FDA approves powder formulation of trofinetide for Rett syndrome. Contemporary Pediatrics. December 12, 2025. Accessed April 7, 2026. https://www.contemporarypediatrics.com/view/fda-approves-powder-formulation-of-trofinetide-for-rett-syndrome