International Rett Syndrome Foundation

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International Rett Syndrome Foundation

IRSF's vision is to create a world without Rett syndrome. We fight for families living with Rett syndrome and a world without it. Learn more at rettsyndrome.org.

Guided by families & experts worldwide, we fund trailblazing research seeking treatments and a cure, empowering support that helps families cope and offers hope, and increasing awareness of Rett. As the leading Rett syndrome research and advocacy organization, the International Rett Syndrome Foundation builds upon our 40-year commitment to breakthrough discoveries and life-changing advancements in

research toward treatments and a cure while supporting families affected by Rett syndrome. Through our legacy foundation pioneers, we have invested over $60M in research leading to identifying Rett syndrome’s cause, demonstrating Rett syndrome is reversible in mice, and supporting the clinical trials that led to the first-ever FDA-approved treatment.

06/26/2026

Today, Acadia Pharmaceuticals announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use has adopted a positive opinion following re-examination, recommending marketing authorization for DAYBU® (trofinetide) for the treatment of neurobehavioral symptoms of Rett syndrome in adults and children ages five and older.

This is a major step forward for Rett syndrome after an initial negative opinion earlier this year, and an important milestone for families in Europe.

The process is not over yet. The recommendation now advances to the European Commission, which is expected to issue a final decision in the coming months. If DAYBU® is approved, the marketing authorization would apply to all 27 EU member states, as well as Iceland, Liechtenstein, and Norway.

IRSF celebrates this progress and is grateful to the families whose participation in research helped make today’s milestone possible.

Read Acadia's Announcement: https://acadia.com/en-us/media/news-releases/daybu-trofinetide-recommended-for-approval-in-the-european-union-by-chmp

06/24/2026

A special shoutout to UCB for joining us as a Gold Sponsor of this year’s ASCEND National Summit!

UCB Biopharma will present during our Clinical Trial Update session on Wednesday morning. Want to learn more, but can't make it to Minnesota next week? We're excited to now offer virtual tickets to livestream the main stage sessions!

Virtual tickets are just $79 through June 30! Learn more today at: rettsyndrome.org/ascend

06/23/2026

At this week's BIO International Convention 2026 in San Diego, IRSF CEO Laura Brod Hameed was invited to serve as a panelist in the Rare Disease Therapeutics track for “Patient Centric Solutions for Clinical Development and Regulatory Pathways.”

Laura attended alongside Immediate Past Board Chair David Pass, PharmD, representing IRSF at one of the biotechnology industry’s most important global convenings.

BIO brings together the global biotech sector with the policy, investment, and industry partnerships that help move innovation into development. It is also a forum for countries that have made biotechnology a national strategy.

Laura’s panel focused on patient-centered approaches to clinical development and regulatory pathways, including how patient and caregiver experience can shape the evidence needed to move potential therapies forward.

For Rett syndrome, this connection is critical. Promising science must be matched with trial strategies, outcome measures, and regulatory pathways that reflect meaningful change for individuals with Rett and their families.

IRSF is committed to bringing Rett syndrome into the scientific and industry conversations shaping the future of rare disease drug development.

06/22/2026

Today, Taysha Gene Therapies shared updates on TSHA-102, its investigational gene therapy for Rett syndrome.

With dosing now complete in the REVEAL pivotal study, Taysha shared additional longer-term efficacy data from Part A of the Phase 1/2 adolescent/adult and pediatric trials. In this part of the trial, 100% of participants gained or regained at least one developmental milestone across the core functional domains of fine motor, gross motor, and communication after receiving TSHA-102, with additional functional gains continuing to accumulate over time through 12 months or longer. TSHA-102 has been generally well tolerated, with no treatment-related serious adverse events or dose-limiting toxicities reported.

We look forward to welcoming the Taysha team to the IRSF Rett Syndrome Scientific Meeting and ASCEND National Summit next week.

Learn More:
🔗 Rett Community Letter:https://tayshagtx.com/wp-content/uploads/Taysha-Rett-Community-Letter-06.22.26.pdf
🔗 Press Release: https://ir.tayshagtx.com/node/10406/pdf

06/21/2026

Happy Father’s Day to the Rett Dads, Stepdads, Grandfathers, and Father-figures who are such an important part of our community.

So much of what you give happens in the moments others may never see — the care built into daily routines, the encouragement through hard seasons, the joy you bring to small victories, and the love that carries your families forward.

Today, we are honored to celebrate you.

Thank you to everyone who shared photos and helped us recognize these incredible men. 💜

06/18/2026

✨New for ASCEND 2026: virtual tickets are now available!

We know travel isn’t always possible, so families can now livestream select ASCEND sessions from home for just $79 when purchased by June 30 ($99 after).

Virtual tickets include access to all main stage sessions and the afternoon breakout track, “Growing into Adulthood.”

💻 Get your virtual ticket to the livestream here: https://cvent.me/4egY8P

For the agenda, speaker list, and more, visit: rettsyndrome.org/ascend.

The ASCEND livestream is powered by RA Capital, Wee Ones Bows, and the IRSF Board of Directors - thank you for helping make livestream access possible for families all over the world.💜

06/17/2026

Father’s Day is just around the corner, and IRSF wants to celebrate the incredible Rett Dads, Stepdads, Grandfathers, and Father-figures in our community!

We are putting together a special video montage to honor these amazing men, and we need your help to make it happen.

How to participate:

📸 Drop your favorite photo in the comments below (or email our Community Engagement Specialist, Megan Ladwig, at [email protected]!)

➕ Optional: Include their first name and a short blurb about what makes them so great!

📅 Deadline: Please submit by Friday, June 19th.

Let’s show our Rett Father-figures some major love. Drop your photos in our inbox today!

(Please note: By submitting your photos, you are giving IRSF permission to share your content on our public social media channels.)

06/12/2026

When Shaimaa found out she was pregnant with twins, she dreamed of having a daughter who would be her best friend and share life with her. Khadija and her twin brother, Tarek, were born, and at first, everything seemed typical.

But as Khadija grew, Shaimaa began noticing developmental delays while Tarek continued developing typically. For years, their family searched for answers, moving between doctors, hospitals, therapy centers, and countless tests. They received different diagnoses along the way, but as Shaimaa shared, “nothing truly explained her condition.”

Then seizures began, bringing emergency hospitalizations, EEGs, MRIs, and genetic testing. Still, answers were hard to find.

Finally, in October 2025, genetic testing revealed that Khadija has Rett syndrome.

“I was devastated,” Shaimaa shared, “but I kept searching for hope.”

That hope began to grow as she learned more about new treatments, ongoing research, and the progress being made for Rett syndrome. Through every difficult moment, Shaimaa says she never stopped believing in her daughter.

Today, with the support of family, friends, and doctors, Shaimaa is holding onto hope more than ever.

As she shared, “I believe that Khadija’s future can be brighter than anyone once thought possible.” ✨

06/11/2026

This week, IRSF’s Director of Translational Research, Bhala Rao, is representing Rett at the World Orphan Drug Congress USA in Boston, where rare disease leaders across research, clinical development, industry, advocacy, and access are coming together to advance progress for rare diseases.

IRSF’s scientific strategy is built to advance Rett research from every angle: investing in critical discovery science, driving therapeutic development, strengthening clinical research and trial readiness, and ensuring that the needs of families are represented as the treatment landscape evolves.

That is why showing up in rooms like this matters. Progress requires strong science, meaningful partnerships, and a clear understanding of what families need today and what they are counting on for the future.

At WODC, Bhala added a message to the “Leave a little hope” wall:

“Rett is rare, but every child is unique. We are in it together.”

We are proud to carry the Rett community into conversations that help shape the future of rare disease research, treatment development, and access. 💜

06/09/2026

This week, we’re highlighting the teams that help turn IRSF’s mission into action behind the scenes. 💜

Growing our impact for families takes more than big ideas. It takes clear communication, strong systems, thoughtful planning, and the day-to-day work that helps every part of IRSF move with purpose.

Right now, much of that work is focused on ASCEND. For months, our Marketing and Operations teams have been helping shape the experience that will welcome families to Minnesota for a week of learning, connection, and community.

From the information you receive before you arrive to the moments you’ll experience onsite, so much care has gone into making this year’s ASCEND meaningful for the families we serve.

We can’t wait to see you next month! Get to know the Marketing and Operations teams helping bring it all together. ✨

Address

Cincinnati, OH

Telephone

+18008187388

Website

https://www.rettsyndrome.org/

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