News
Latest news
Stories, statements and updates from Rett Syndrome Europe and our member associations across Europe.
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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 trial exceeded its target, treating 25 participants due to high demand from the community.
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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 how families can take part.
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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.
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Rett Syndrome Europe at ECRD 2026: Advancing Visibility, Data, and Care
Rett Syndrome Europe took part in ECRD 2026 with two posters — the rettX patient registry and a study on unmet needs and fragmented care across Europe.
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Statement from Rett Syndrome Europe
Regarding the CHMP Opinion on Trofinetide The European Medicines Agency (EMA) has announced that its Committee for Medicinal Products for Human Use (CHMP) has recommended not granting marketing authorisation for Trofinetide for the treatment of Rett syndrome in Europe. According to EMA, the CHMP concluded that the treatment effects observed after 12 weeks were not sufficient…
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International Rett Syndrome Conference – Istanbul, Turkey 11th – 12th October 2025
Registration is now open for the International Rett Syndrome Conference in Istanbul! A unique gathering for doctors, scientists, families, and therapists! Be a part of this important event and stay up to date with the latest scientific developments in the Rett syndrome field! Register here now! The International Rett Syndrome Conference 2025 is now open…
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Taysha share more information on Part A and Part B of their gene therapy trial
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.
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Family Focus Story
To mark Rare Disease Day 2025 we are sharing the amazing adventures of Selena – an very inspiring young lady with Rett syndrome and her equally wonderful family!
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RSE February 2025 E Newsletter
Research updates, events and family focus stories plus an opportunity to hear from Tom Pulles and Stephanie Kim from Acadia on 4th March 2025, 7pm CET – you will need to register first to join the webinar.
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Latest Update From Taysha
We are delighted to share the very positive update from Taysha on their gene therapy programme for Rett syndrome. Ten participants have now received the therapy which has generally been well tolerated with no serious adverse side effects. They are in discussion with the FDA about moving to the next phase which is dosing more…
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