
More details on the changes seen so far in the first cohort of individuals who received either the high or low dose, ages 6-21 years. No serious adverse events seen at either dose. Read the full response below.
More details on the changes seen so far in the first cohort of individuals who received either the high or low dose, ages 6-21 years. No serious adverse events seen at either dose. Read the full response below.

More details on the changes seen so far in the first cohort of individuals who received either the high or low dose, ages 6-21 years. No serious adverse events seen at either dose. Read the full response below.

Neurogene has announced the successful completion of enrollment in the EMBOLDEN trial of NGN-401, an investigational gene therapy for Rett syndrome. The…

rettX is the pan-European, patient-led Rett syndrome registry led by Rett Syndrome Europe, presented at ECRD 2026. Learn what it is and…

A multi-country caregiver study presented by Rett Syndrome Europe at ECRD 2026 reveals consistent unmet needs and fragmented care across Europe.