Industry News Archives | International Rett Syndrome Foundation

March 31, 2025

European Commission grants NTI64 Orphan Drug Designation for Rett Syndrome

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Taysha

Taysha Gene Therapies Announces Regenerative Medicine Advanced Therapy (RMAT) Designation Granted by U.S. FDA for TSHA-102 in Rett Syndrome

Acadia

Acadia Pharmaceuticals Announces Trofinetide New Drug Submission for Treatment of Rett Syndrome Has Been Accepted for Filing and Priority Review by Health Canada

Unravel

Unravel Announces Initiation of RVL001 Clinical Trial Material Manufacturing to Support Upcoming US and International Clinical Trials in Rett Syndrome

Taysha

Taysha Shares Interim Clinical Data from Cohort One on FY23 Financial Results Call

DepYmed

DepYmed Receives Clearance from U.S. Food and Drug Administration to Initiate Phase 1 Clinical Trial for DPM-1003 for the Treatment of Rett Syndrome

Neurotech

All Phase I/II Rett Syndrome Patients to Receive NTI164 Treatment for a Total of 52 Weeks

Neurogene

Neurogene Announces Expansion and Plans for More Rapid Patient Enrollment of Rett Syndrome Gene Therapy Clinical Trial

Taysha

Taysha Gene Therapies Announces Updates to TSHA-102 Clinical Program in Rett Syndrome

Taysha

Taysha Gene Therapies Announces First Pediatric Patient Dosed with TSHA-102 in REVEAL Phase 1/2 Pediatric Trial in Rett Syndrome