Industry News Archives | International Rett Syndrome Foundation

June 30, 2025

Neurogene Announces Registrational Trial Design for Embolden™ Study of NGN-401 Gene Therapy for Rett Syndrome (Community Letter)

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Acadia

Clinical Data from Open-Label DAFFODIL™ Study Evaluating Long-term Safety of DAYBUE® (trofinetide) in Patients with Rett Syndrome Published in Med

Minoryx

Minoryx announces dosing of the first patient with leriglitazone in the phase 2a study in Rett syndrome (TREE study)

Taysha

Taysha Gene Therapies Reports Full-Year 2024 Financial Results and Provides Corporate Update

Acadia

Acadia Pharmaceuticals Submits Marketing Authorization Application to the European Medicines Agency for Trofinetide for the Treatment of Rett Syndrome

Neurotech

Neurotech Receives US Orphan Drug Designation in Rett Syndrome

Neurogene

Update on Patient SAE & Discontinuation of High Dose Protocol (Community Letter)

Neurogene

Update on Patient SAE (Community Letter)

Neurogene

Neurogene Provides Update on NGN-401 Gene Therapy Clinical Trial for Rett Syndrome

Taysha

Taysha Gene Therapies Reports Third Quarter 2024 Financial Results and Provides Corporate Update