News & Updates
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IRSF Blog
Explore our blog for inspiring stories and helpful information about Rett syndrome. Our posts focus on the human side of Rett, sharing personal experiences and insights to support families and individuals in the Rett community.
Taysha Gene Therapies Updates Community on Positive Initial Findings and Expansion of TSHA-102 Clinical Trial
Today, Taysha Gene Therapies made several exciting announcements about the progress of their REVEAL Phase 1/2 trial in Canada and the future study of their gene therapy, TSHA-102. To read […]
Industry News
Be the first to hear about developments in the Rett syndrome pipeline. Stay informed with essential updates on research, treatments, clinical trials, and more—all tailored to you.
- 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
- 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
- Neurotech
Neurotech Receives US Orphan Drug Designation in Rett Syndrome
- Neurogene
Update on Patient SAE & Discontinuation of High Dose Protocol (Community Letter)
- Neurogene
Neurogene Provides Update on NGN-401 Gene Therapy Clinical Trial for Rett Syndrome
- Neurogene
Update on Patient SAE (Community Letter)
- 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
- 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
- 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
- Anavex
Anavex Announces Topline Results from Phase 2/3 EXCELLENCE Clinical Study in Pediatric Rett Syndrome
- Neurogene
Neurogene Announces Closing of Merger with Neoleukin Therapeutics and Concurrent Private Placement of $95 Million
- Neurogene
Neurogene Doses First Patients in Phase 1/2 Trial of NGN-401 for the Treatment of Female Pediatric Patients with Rett Syndrome
- Taysha
Taysha Gene Therapies Announces Expanded Eligibility in REVEAL Phase 1/2 Adult Trial to Include Adolescent Rett Syndrome Patients
- Taysha
Early, interim data from the first two adult patients dosed shared in Taysha Q3 Financials
- Lucy
Lucy Therapeutics Expands Cutting-Edge Medical Research Program
- Taysha
Taysha Gene Therapies Announces Second Patient Dosed with TSHA-102 in the REVEAL Phase 1/2 Adult Trial for the Treatment of Rett Syndrome
- Neurotech
Neurotech Completes Patient Recruitment in Phase I/II Rett Syndrome Clinical Trial
- Taysha
Taysha Gene Therapies Announces Fast Track Designation Granted by U.S. FDA for TSHA-102 in Rett Syndrome
- Taysha
Taysha Gene Therapies Reports Initial Clinical Data from First Adult Rett Syndrome Patient Dosed in REVEAL Phase 1/2 Trial and Provides Corporate Update with Second Quarter 2023 Financial Results
- Taysha
Taysha Gene Therapies Announces Positive Recommendation from Independent Data Monitoring Committee of REVEAL Phase 1/2 Trial in Rett Syndrome
- Acadia
Acadia Pharmaceuticals Acquires Ex-North American Rights to Trofinetide and Global Rights to Neuren’s NNZ-2591 in Rett Syndrome and Fragile X Syndrome
- Anavex
Long-term Clinical Study Demonstrates Disease-Modifying Effects of ANAVEX®2-73 for Rett Syndrome
- Taysha
Taysha Gene Therapies Provides Clinical Updates for Investigational Programs TSHA-120 in Giant Axonal Neuropathy (GAN) and TSHA-102 in Rett Syndrome at R&D Day
- Neurogene
Neurogene Opens Enrollment for U.S. Gene Therapy Clinical Trial (Community Letter)
- Anavex
Completion of ANAVEX®2-73 (blarcamesine) EXCELLENCE Phase 2/3 Rett Syndrome Clinical Trial
- Taysha
Taysha Gene Therapies Announces First Patient Dosed with TSHA-102 in the REVEAL Phase 1/2 Trial Under Investigation for the Treatment of Rett Syndrome
- Acadia
Acadia Pharmaceuticals Announces DAYBUE™ (trofinetide) is Now Available for the Treatment of Rett Syndrome
- Neurotech
Neurotech to Launch Phase II Clinical Trial in Rett Syndrome
- Acadia
Acadia Pharmaceuticals Announces U.S. FDA Approval of DAYBUE™ (trofinetide) for the Treatment of Rett Syndrome in Adult and Pediatric Patients Two Years of Age and Older
- Acadia
LILAC-1 Study Topline Results Announced (Community Letter)
- Unravel
Unravel Biosciences Announces Positive Feedback from FDA Pre-IND Meeting on RVL001 Rett Syndrome Program
- Anavex
ANAVEX®2-73 (Blarcamesine) AVATAR Phase 3 Trial met Primary and Secondary Efficacy Endpoints
- Taysha
Taysha Gene Therapies Provides Update on TSHA-120 Program in Giant Axonal Neuropathy and a 2023 Corporate Outlook
- Neurogene
Neurogene Announces FDA Clearance of IND for NGN-401 Gene Therapy for Children with Rett Syndrome
- Taysha
Astellas and Taysha Gene Therapies Announce Strategic Investment to Support Development of Taysha’s AAV-based Gene Therapy Programs
- Acadia
Acadia Pharmaceuticals Announces Trofinetide New Drug Application for the Treatment of Rett Syndrome has been Accepted for Filing and Review by U.S. FDA
- Acadia
Acadia Pharmaceuticals Submits New Drug Application to the U.S. FDA for Trofinetide for the Treatment of Rett Syndrome
- Alcyone
Alcyone Presents Preclinical Data on Gene Therapy Programs with The Center for Gene Therapy at The Abigail Wexner Research Institute (AWRI) at ASGCT 2022
- Taysha
Taysha Gene Therapies Announces Initiation of Clinical Development of TSHA-102 in Rett Syndrome
- Stoke Acadia
Acadia Pharmaceuticals and Stoke Therapeutics Announce Collaboration to Pursue Multiple RNA-based Treatments for Severe and Rare Genetic Neurodevelopmental Diseases
- Acadia
Acadia Pharmaceuticals Announces Positive Top-line Results from the Pivotal Phase 3 Lavender Trial of Trofinetide in Rett Syndrome
- Anavex
Anavex Life Sciences Announces ANAVEX®2-73 Meets Primary and Secondary Endpoints in Clinical Trial
- Anavex
Anavex Life Sciences Announces First Patient Dosed in Phase 2 Clinical Trial of ANAVEX®2-73
- AMO
AMO Pharma Receives FDA Orphan Drug Designation for AMO-04 in Treatment of Rett Syndrome
- Neurolixis
European Commission approves Orphan Medicinal Product designation for NLX-101
- Neurolixis
European Commission approves Orphan Medicinal Product designation for NLX-101
Press Releases
Read official announcements from IRSF, covering the latest initiatives, research partnerships, and community events related to Rett syndrome.
2024 Edition
IRSF Rett Gazette
The Rett Gazette is IRSF’s annual fall publication providing updates and information to the Rett syndrome community. Each year, the Gazette covers various topics related to Rett syndrome, such as advancements in research, real-world experiences with treatments, advocacy efforts, and personal stories from the community.
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