
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.
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.

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.

Rett Syndrome Europe welcomes the positive CHMP opinion for Daybu (trofinetide), a meaningful milestone for families affected by Rett syndrome across Europe.

Taysha Gene Therapies has shared positive longer-term interim data from its investigational gene therapy TSHA-102, with dosing now complete in the pivotal…

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