News from the Rett Syndrome Pipeline
Press releases, community letters, and more from the companies advancing towards treatments and cures for Rett syndrome.
June 19, 2025
Taysha Gene Therapies Updates on Part A Data, Plans for the Part B Pivotal Study, and FAQs (Community Letter)
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- 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
- Neurogene
Neurogene Announces First Patient Dosed in High-Dose Cohort of NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
- 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 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
- Taysha
Taysha Gene Therapies Announces Pivotal Part B Trial Design Details for TSHA-102 in Rett Syndrome Enabled by IRSF Natural History Data and Positive Clinical Data from Part A of the REVEAL Adult/Adolescent and Pediatric Trials Evaluating TSHA-102
- Acadia
Acadia Pharmaceuticals Announces Recipients of Second Annual Rett Sibling Scholarship
- Taysha
Taysha Gene Therapies Reports First Quarter 2025 Financial Results and Provides Corporate Update
- Neurotech
European Commission grants NTI64 Orphan Drug Designation for Rett Syndrome
- Neurogene
Neurogene Reports Fourth Quarter and Full Year 2024 Financial Results and Highlights Recent Updates
- 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
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