You can read the full press release here . They expect it will be 10-14 months before a decision is known. You might also like to read this plain language summary Trofinetide Treatment Demonstrates a Benefit Over Placebo for the Ability to Communicate in Rett Syndrome.
You can read the full press release here . They expect it will be 10-14 months before a decision is known.
You might also like to read this plain language summary Trofinetide Treatment Demonstrates a Benefit Over Placebo for the Ability to Communicate in Rett Syndrome.
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.