Shots:
The CHMP has recommended Daybu (trofinetide) for the treatment of neurobehavioral symptoms of Rett syndrome in pts (≥5yrs.); If approved, authorization would be valid in all 30 EEA states
Opinion was based on the results from the P-III (LAVENDER) study, which showed improvements in core features of Rett syndrome, as measured by RSBQ & CGI-I…
Shots:
Neurogene has reported interim data from the pediatric cohort (4-10yrs.) of the ongoing P-I/II trial assessing NGN-401 for the treatment of females with Rett syndrome
As of Oct 30, 2025, all pediatric pts (n=8) had functional gains across Rett syndrome domains, achieving 35 developmental milestones in fine motor, communication & ambulation. These gains were…

