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Hopeful Progress: Gene Therapy Trial for Rett Syndrome Completes Enrollment
Neurogene has announced the successful completion of enrollment in the EMBOLDEN trial of NGN-401, an investigational gene therapy for Rett syndrome. The…
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rettX: Building a Patient-Led Rett Syndrome Registry for Europe
rettX is the pan-European, patient-led Rett syndrome registry led by Rett Syndrome Europe, presented at ECRD 2026. Learn what it is and…
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Consistent Unmet Needs and Fragmented Care in Rett Syndrome Across Europe
A multi-country caregiver study presented by Rett Syndrome Europe at ECRD 2026 reveals consistent unmet needs and fragmented care across Europe.