Industry News Archives | International Rett Syndrome Foundation

March 31, 2025

European Commission grants NTI64 Orphan Drug Designation for Rett Syndrome

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Acadia

Acadia Pharmaceuticals Presents DAYBUE™ (trofinetide) Real-World Evidence and Additional Data in Rett Syndrome at the 2024 International Rett Syndrome Foundation (IRSF) Annual Scientific Meeting

Neurogene

Neurogene Announces First Patient Dosed in High-Dose Cohort of NGN-401 Gene Therapy Clinical Trial for Rett Syndrome

Neurogene

Neurogene Announces NGN-401 Gene Therapy for Rett Syndrome Selected by FDA for START Pilot Program

Acadia

A Letter to the Rett Community with Updates on DAYBUE

Unravel

Unravel Biosciences Receives FDA Orphan Drug Designation for Vorinostat (RVL-001) as a Treatment for Rett Syndrome

Unravel

Unravel Announces Collaboration with Vanderbilt University Medical Center to Support Upcoming Multicenter U.S. Clinical Study in Rett Syndrome that Includes RVL-001

Neurogene

Neurogene Receives Australian HREC Approval for Trial

Neurogene

Neurogene Presents Favorable Safety Data from Phase 1/2 Trial of NGN-401 Gene Therapy for Rett Syndrome during ASGCT Annual Meeting

Neurotech

Neurotech Reports Significant Clinical Benefits and Safety in Phase I/II Rett Syndrome Clinical Trial