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 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
- 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
- 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 Rett Sibling Scholarship Applications Now Open (Community Letter)
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