New Drug Shows Promise for Treating Rett Syndrome

Rett Syndrome is a rare genetic neurological disorder that affects mostly female children (1 in 10,000 births) and is associated with significant impairments in nearly every aspect of life. It is characterized by developmental regression, loss of language and hand function, impaired gait, and near constant repetitive hand movements while awake.[0] Seizures, scoliosis, breathing issues, and gastrointestinal problems are also common. Rett Syndrome is usually recognized between 6 to 18 months, when the child begins to miss developmental milestones or loses abilities they had previously gained. There is currently no treatment available for Rett Syndrome, however, the drug Trofinetide is showing promising results in clinical trials, having been found to improve breathing irregularities and reduce hand and body movements in Rett Syndrome patients.[1] If approved, Trofinetide would be the first drug to treat any neurodevelopmental disorder, and the first New Zealand-discovered neurological drug to be cleared by the major regulator.[0] Trofinetide is designed to reduce neuroinflammation and support synaptic function, thus addressing the core symptoms of Rett Syndrome.[2] Pre-clinical studies in rat models and later clinical trials conducted in children have both confirmed that Trofinetide is safe, well-tolerated, and brings improvements in symptoms for Rett Syndrome.[3]

0. “Acadia Awaits Potential Approval of First Rett Syndrome Medicine” BioSpace, 10 Mar. 2023,

1. “International Rett Syndrome Foundation Celebrates FDA Approval of Trofinetide for Treatment of Rett Syndrome” EIN News, 11 Mar. 2023,

2. “Acadia wins FDA nod for Rett syndrome therapy (NASDAQ:ACAD)” Seeking Alpha, 11 Mar. 2023,

3. “Will NZ neurological drug Trofinetide for Rett Syndrome get US FDA approval and make history?” New Zealand Herald, 9 Mar. 2023,

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