Neuren’s trofinetide successful in Phase 2 clinical trial in Fragile X

Neuren’s trofinetide successful in Phase 2 clinical trial in Fragile X

We are pleased to share great news adapted from Neuren’s press release: Neuren’s phase 2 trial has successfully established proof of concept and provides a strong rationale for Neuren to move forward with developing trofinetide for Fragile X syndrome. In this initial small trial with a relatively short treatment period, trofinetide was very well tolerated, with the high dose (70 mg/kg twice daily) demonstrating a consistent pattern of clinical improvement, observed in both clinician and caregiver assessments. After only 28 days of treatment, improvements were seen across core symptoms of Fragile X syndrome, including higher sensory tolerance, reduced anxiety, better self-regulation and more social engagement. No serious adverse events were reported. Positive Results in Fragile X Syndrome and Rett Syndrome Beneficial effects of trofinetide have now been observed in both Fragile X syndrome and Rett syndrome. Neuren announced encouraging results of their phase 2 trofinetide trial in Rett Syndrome last year. Trofinetide Tested at FRAXA’s Drug Validation

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