The USFDA has recently approved the first treatment, trofinetide, for Rett syndrome. The approval of trofinetide is based on a 12-week study with adult and pediatric patients (Table 1) and another study with only pediatric patients. About Rett syndrome A rare genetic disorder Characterized by gradual loss of motor skills and language Hallmark: Constant repetitive hand movements, including clapping or rubbing Clinical trials Table 1: Study details In an open-label study with patients between two and four years of age, 13 patients received trofinetide for at least 12 weeks, and nine patients received trofinetide for at least six months.
Patients exhibited similar adverse reactions as in the abovementioned study. Designations received : Priority review, orphan drug, and fast track Indication: For patients with Rett syndrome who are two years of age and older Administration The recommended…
