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 Reports Third Quarter 2024 Financial Results and Provides Corporate Update

Neurogene

Neurogene Community Letter

Neurogene

Neurogene Reports Positive Interim Efficacy Data from First Four Low-Dose Pediatric Participants in NGN-401 Gene Therapy Clinical Trial for Rett Syndrome

Acadia

Acadia Pharmaceuticals Announces Health Canada Approval of DAYBUE™ (trofinetide) for the Treatment of Rett Syndrome

Acadia

Acadia Rett Sibling Scholarship Applications Now Open (Community Letter)

Neurotech

Neurotech Files for US Orphan Drug Designation in Rett Syndrome

Neurogene

Neurogene Announces RMAT Designation for NGN-401 Investigational Gene Therapy for Rett Syndrome

Acadia

Clinical Data from Open-Label Extension LILAC-1™ and LILAC-2™ Studies Evaluating Long-Term Safety and Efficacy of DAYBUE™ (trofinetide) in Patients with Rett Syndrome Published in Med

Taysha

Taysha Gene Therapies Announces Positive Clinical Data Across Adult and Pediatric Patients from Low Dose Cohort in Ongoing REVEAL Phase 1/2 Trials Evaluating TSHA-102 in Rett Syndrome