Ann: Rett Syndrome Phase I/II trial shows significant benefits, page-6

It seems that there is some potential synergy in communication between NTI164 which was strong in verbal comm, and Daybue which was strong in communication choices, but weak in verbal! Maybe this is just randomness. In the second quote, caregivers seem not to mention anxiety and body rocking. So there may be some synergies between Daybue and NTI164.


Acadia/Neuren haven't released much on the make up of the CGI-S results. See link under the quote:

Daybue communication results: "
Results

Trofinetide demonstrated a statistically significant difference versus placebo for the CSBS-DP-IT Social Composite score (least squares mean [LSM] difference = 1.0; 95% confidence interval [CI], 0.3 to 1.7; P = 0.0064; Cohen's d effect size = 0.43) and a nominally significant difference for the RTT-COMC (LSM difference: −0.3; 95% CI, −0.6 to −0.0; P = 0.0257; Cohen's d effect size = 0.36). As expected, there was no difference for the RTT-VCOM."

https://www.sciencedirect.com/science/article/pii/S0887899423004058



"27 caregivers of 26 study participants took part in optional exit interviews to explore the patients’ experiences with Rett syndrome and DAYBUE during the trials. The most frequently reported improvements observed by caregivers to participants treated with DAYBUE in the studies were engagement with others (46.2%), hand use (42.3%), and eye gaze (30.8%). Caregivers also reported that some participants acquired new sounds (23.1%) or words (19.2%)."

https://acadia.com/media/news-relea...ide-in-patients-with-rett-syndrome-published/