News & Updates
Be the first to know about developments in Rett syndrome. Stay informed on research, treatments, events, and more with updates that matter to you.
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.
Raising a Hand: A Journey of Healing, Hope, and Friendship
Inconceivable. That’s the word that photographer Dave Clements uses to describe the emotion swelling in his…
DAYBUE in the Real World
Answers to your most frequently asked questions and resources to find out more about the first-ever…
Transforming the Landscape of Rett Research
The International Rett Syndrome Foundation (IRSF) hosted its annual scientific meeting in Westminster, Colorado on June…
Spotlight on Communication: An Interview with Dr. Theresa Bartolotta
For Theresa Bartolotta, Ph.D., CCC-SLP, good things have come through community. Bartolotta, a speech-language pathologist (SLP)…
Advocating for YOU: Rare Disease Week on Capitol Hill
IRSF works tirelessly to advocate for you and your loved one with Rett syndrome. Our work…
Neurogene Announces Expansion of Gene Therapy Clinical Trial
Today, Neurogene announced plans to expand and more rapidly enroll patients their Phase 1/2 clinical trial…
Taysha Gene Therapies Adolescent & Adult Study Expanded to U.S.
Today, Taysha Gene Therapies shared several exciting updates on their REVEAL Phase 1/2 clinical trial of…
My Rett Ally: Revolutionizing Care Management for Rett Families
On a recent visit to the emergency room, it took just seconds for Samantha Brant to…
Taysha Gene Therapies Doses First Pediatric Patient in U.S. Gene Therapy Clinical Trial
Taysha Gene Therapies today announced the dosing of the first pediatric patient in their REVEAL Phase…
Neurogene Announces First Pediatric Patients Dosed in US Gene Therapy Clinical Trial
We’re excited to share that today Neurogene announced that two pediatric patients have been dosed in…
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…
Acadia Pharmaceuticals Acquires International Rights to Trofinetide
BIG NEWS for our international Rett syndrome community! Today, Acadia Pharmaceuticals announced they have acquired the…
RettAway Reflections
By Samantha Brant, Family & Community Engagement Manager, and mom to Macy As I returned home…
Clinical Trial Updates from Taysha & Anavex
We are excited to share news from two pharmaceutical companies working on potential treatments and cures…
Neurogene Announces Locations & Opens Enrollment for US Gene Therapy Clinical Trial
We’re excited to share that today Neurogene announced the locations and began enrolling for the first…
A First for Rett: FDA Approves Trofinetide for Treatment of Rett Syndrome!
This first-ever treatment is approved for individuals with Rett syndrome. (Updated March 19, 2023) On March…
Neurogene Announces First Rett Syndrome Gene Therapy Trial in U.S.
We’re excited to share that Neurogene announced today that the FDA approved their Investigational New Drug…
Flu, COVID, & RSV: A Message to our Rett Community
By Dr. Tim Benke, IRSF Medical Advisor As many of you know, the United States is…
Taysha Begins Recruitment for Gene Therapy Trial
Next Huge Milestone for Rett The last 12 months have been an unprecedented time for Rett…
Your Voice Matters to the FDA & Pharmaceutical Industry
By Dr. Dominique Pichard, IRSF CSO – The patient voice has increasingly become a focus for…
FDA Grants Trofinetide Priority Review
ACADIA has shared the exciting news that the US Food and Drug Administration (FDA) has reviewed…
In-Person Strollathons Are Back!
In September IRSF returns with our in-person Strollathons! These nationwide fundraisers bring together local communities to…
IRSF Expands Center of Excellence Network
The International Rett Syndrome Foundation is expanding our community’s access to best-in-class care by adding four…
The Summit of Hope and Healing: ASCEND 2022 Rett Syndrome National Summit
By Katie Busch, IRSF Program Coordinator – During the last week of April 2022, the International…
Accelerating Discoveries in Rett Research: 2022 IRSF Rett Syndrome Scientific Meeting
In April 2022, the International Rett Syndrome Foundation hosted the IRSF Rett Syndrome Scientific Meeting in…
Taysha Rett Syndrome Community Update
From IRSF: 2022 is a big year for Rett syndrome: the return of an in-person Family…
IRSF Sets Up Next Wave of Rett Breakthroughs with $2.3 M Research Investment
Dr. Dominique Pichard, IRSF Chief Science Officer Get ready for the next wave of research targeting…
Positive Top-line Results for Phase 3 Trofinetide Trial
Melissa Kennedy, Chief Executive Officer On December 6, the Rett syndrome community received some very encouraging…
Help Unlock Treatments & Cures for Rett with IRSF’s Rett Syndrome Registry
Your family can be the key that researchers and industry need to create a world without…
International Rett Syndrome Foundation’s New Center of Excellence Network
The International Rett Syndrome Foundation is committed to advancing and expanding our community’s access to best-in-class…
Kelsey & Toyua: Friendship & Hope Just 20 Minutes Away
Kelsey Clark felt alone and isolated when her five-year-old daughter Charlee was diagnosed with Rett syndrome…
Board of Directors Welcomes Alan Shukovsky —The Father of a Boy Living With Rett
When Alan Shukovsky and his wife, Eva, were getting married, their rabbi told them that it…
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.
- Acadia
Acadia Pharmaceuticals Submits Marketing Authorization Application to the European Medicines Agency for Trofinetide for the Treatment of 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
- 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
- Acadia
A Letter to the Rett Community with Updates on DAYBUE
- Neurogene
Neurogene Announces NGN-401 Gene Therapy for Rett Syndrome Selected by FDA for START Pilot Program
- 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.
IRSF Leadership Update
IRSF Welcomes Two to Board of Directors
The International Rett Syndrome Foundation (IRSF), a leading patient advocacy foundation dedicated to funding research and…
- Clinical, Research
Center of Excellence Clinic Network Expanded
Three additional clinics recognized for best-in-class Rett syndrome care. The International Rett Syndrome Foundation (IRSF) proudly…
- Clinical, Research
IRSF Announces Expansion of Scientific Leadership
The new leadership appointments include a key promotion and new hire to advance research and clinical…
Introducing My Rett Ally: A New Tool to Support Caregivers
My Rett Ally, a web application powered by mejo, is specifically designed for Rett syndrome patients…
IRSF Welcomes Three New Members to its Board of Directors
The International Rett Syndrome Foundation (IRSF), a mission-driven advocacy foundation focused on funding research and supporting…
- Acadia, Clinical, Research
IRSF Establishes Research Fund for Males with Rett Syndrome
The fund is established thanks to an inaugural gift from the Otis Family in honor of…
IRSF Awards $4.4M in Rett Syndrome Research Grants
Our commitment to setting up the next wave of Rett breakthroughs continues with the largest annual…
IRSF Receives $500,000 Donation from Vezbi Super App
International Rett Syndrome Foundation (IRSF) is proud to announce that it is the recipient of a…
IRSF Launches Clinical Trial Committee to Support the Development of Rett Syndrome Treatments
The Clinical Trial Committee includes 14 leading U.S. Rett syndrome specialists CINCINNATI – (May 31, 2022)…
Federal Funding Bill for 2022 Supports Research for Rett Syndrome
CINCINNATI – March 22, 2022 – The Omnibus Fiscal Year 2022 (FY22) appropriations bill passed by…
Hive Networks & IRSF Partner to Advance Research and Treatment for Rett Syndrome
Hive Networks today announced a multi-year partnership with the International Rett Syndrome Foundation to provide a…
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.
Stay Current on Rett
Join our community and receive regular updates on Rett syndrome news, breakthroughs, and new resources, tailored for those impacted by Rett.