FRAXA Research Foundation

06/04/2026

We’re less than one week away from FRAXA’s next community webinar: The Fragile X Treatment Pipeline: Current and Emerging Clinical Trials.

Join us on Tuesday, June 10 at 5:00 PM Eastern for a practical, forward-looking discussion on Fragile X clinical trials that are underway or coming soon.

Featuring leading Fragile X clinical researchers Elizabeth M. Berry-Kravis, MD, PhD, and Craig Erickson, MD, this session will cover current and emerging programs from Spinogenix, Mirum, Servier, and others.

Hosted by FRAXA’s Mike Tranfaglia, MD, the conversation will focus on where each program stands today, the science behind these approaches, and what families may want to know about eligibility and participation.

A live Q&A will follow the presentations.

Register today to join us.

Join FRAXA Research Foundation for a community webinar on the Fragile X treatment pipeline, featuring leading Fragile X clinical researchers Elizabeth M. Berry-Kravis, MD, PhD, and Craig Erickson, MD.

05/27/2026

FRAXA's first Scandinavian research grant has been awarded to Francesco Longo, PhD, and Charlène Perian, PhD, at University of Gothenburg in Sweden. This $100,000 award will fund studies on how defects in the autophagy-lysosome pathway may contribute to altered dopamine signaling and behavioral inflexibility in Fragile X syndrome.

Using mouse models, the team will test whether activating the master protein regulator TFEB can restore dopamine function and improve behavioral flexibility. This work could help validate a new therapeutic approach for Fragile X syndrome.

FRAXA-funded researchers at Gothenburg University study whether activating TFEB can restore dopamine function and improve flexibility in Fragile X syndrome.

05/22/2026

For 30 years, Patrick's PALS has been so much more than a basketball tournament. 🩵🏀

What started in 1997 as a group of friends wanting to help honor Patrick Vershbow and support Fragile X research has grown into a powerful tradition of community, friendship, awareness, and hope.

This slideshow is a look back at 29 incredible years of memories, laughter, competition, and love. From the early days in Cambridge gyms to today at the incredible TRACK at New Balance, one thing has remained the same: people coming together to make a difference.

Because of this amazing community, Patrick’s PALS has raised more than $2.5 million for FRAXA to fund research to find effective treatments and, ultimately, a cure for Fragile X syndrome.

Most importantly, this event continues because of the generous, loyal, compassionate people who believe in this mission and in families like the Vershbows.

To everyone who has been part of Patrick’s PALS over the last 30 years - Thank You! 🩵

Now it’s time for Year 30, and we would love for YOU to be part of it!!

📍 Patrick’s PALS 30
📅 Saturday, May 30, 2026
📌 TRACK at New Balance
91 Guest Street, Brighton, MA

Come watch great basketball, enjoy raffles, silent auction items, food, contests, ice cream, and an incredible day filled with community and purpose.

Whether you’ve been there since Year 1 or this will be your first time attending, there’s a place for you at Patrick’s PALS.

Let’s celebrate 30 years of Patrick's PALS together and continue advancing Fragile X research.

05/21/2026

What’s Next in Fragile X Clinical Trials?

Join FRAXA Research Foundation on Tuesday, June 10 at 5:00 PM Eastern for a practical, forward-looking webinar on the Fragile X treatment pipeline.

Featuring Elizabeth M. Berry-Kravis, MD, PhD, and Craig Erickson, MD, this session will explain current and emerging clinical trials for Fragile X syndrome, including programs from Spinogenix, Mirum, Servier, and others. The discussion will cover the science behind these approaches, where each program stands today, and what families may want to know about eligibility and participation.

A live Q&A will follow the presentations.

Join FRAXA Research Foundation for a community webinar on the Fragile X treatment pipeline, featuring leading Fragile X clinical researchers Elizabeth M. Berry-Kravis, MD, PhD, and Craig Erickson, MD.

05/19/2026

The recording is now available from our community webinar with Shionogi and The National Fragile X Foundation that happened earlier today.

In this conversation, Shionogi discusses topline results of the EXPERIENCE clinical trial program evaluating zatolmilast in Fragile X syndrome, including what is known so far, the ongoing open-label extension, and what comes next as they continue analyzing the data. Questions from the community were addressed.

FRAXA Research Foundation and the National Fragile X Foundation hos...

05/18/2026

For 30 years, Patrick's PALS has shown what sustained community commitment can do for Fragile X research.

Their 30th annual 3-on-3 Basketball Tournament is more than a tradition. It reflects three decades of people coming together for a great day aimed at one goal: better treatments and a cure for Fragile X syndrome.

At FRAXA Research Foundation, that kind of long-term partnership matters. Progress in biomedical research takes persistence, and support like this helps drive the advances families are waiting for.

We’re grateful to Patrick’s PALS for standing with the Fragile X community over the long haul and helping keep Fragile X syndrome research moving forward.

Watertown-based Patrick’s PALS is in its 30th year, raising money for research into Fragile X Syndrome, the most common inherited cause of autism and intellectual disabilities. A 3-on-3 basketball fundraiser will be held at the TRACK at New Balance. See more details provided by Patrick’s PALS, b...

05/15/2026

Clinical Trials Arena has published a recap of Shionogi’s update on zatolmilast in Fragile X syndrome.

The article summarizes the Phase 3 EXPERIENCE program, including the primary endpoint misses, the signal seen in the adult study, and Shionogi’s plan to continue the open-label extension while further evaluating the data.

For the Fragile X community, this remains a difficult setback. It also underscores why better outcome measures, thoughtful clinical trial design, and continued investment in Fragile X research are so important.

FRAXA remains committed to learning from every trial and helping drive the next advances toward effective treatments and ultimately a cure.

Shionogi's zatolmilast demonstrated an efficacy signal in one FXS study in adults, but it missed its primary endpoints across the programme.

05/15/2026

⛳ We are just ONE WEEK away from the 2026 FRAXA Charity Golf Tournament!

For almost years, the Hall family and friends have helped raise more than $465,000 for FRAXA Research Foundation in support of Fragile X research and this year, they are aiming to SHATTER the half-million-dollar mark! 💙

The tournament will feature great contests, incredible auction items, food, fun, and a wonderful day of community all while helping advance research toward effective treatments and a cure for Fragile X syndrome.

A huge thank you to Pete, Dave, Jake, and Sam Hall, along with Jack Murphy, for their dedication, hard work, and continued commitment to families living with Fragile X syndrome. Your passion and generosity continue to make an incredible impact.

📍 Friday, May 22, 2026
📍 Bradford Country Club – Haverhill, MA
⛳ 8:00 AM Shotgun Start

Learn more or register at https://www.fraxa.org/event/2026-fraxa-charity-golf-tournament/

05/14/2026

Do you have questions after yesterday’s Shionogi update on the Phase 3 zatolmilast trials in Fragile X syndrome?

FRAXA and The National Fragile X Foundation will host Shionogi for a community webinar on May 19 at 12:00 PM Eastern to talk through the update, share what is known so far, and discuss what comes next.

Attendees may submit questions when registering, and earlier submissions are more likely to be addressed during the conversation. The webinar will be recorded and made available shortly afterward for those who are unable to attend live.

Join FRAXA, the National Fragile X Foundation, and Shionogi for a community webinar on the EXPERIENCE trial update in Fragile X syndrome.

05/13/2026

Shionogi has shared an important update on its Phase 3 clinical trials of zatolmilast in Fragile X syndrome.

Neither trial met its original endpoint. In the adult study, there was a statistically significant signal on the Numeric Rating Scale, a caregiver assessment that includes language use, but because it was a secondary measure, that finding alone is not typically enough to support FDA approval.

This is a painful setback for families and for everyone working toward effective treatments for Fragile X syndrome. It is also a reminder of how urgently the field needs better clinical trial methods, stronger outcome measures, and continued investment in Fragile X research.

At FRAXA, we are committed to learning everything we can from this experience and using those lessons to help drive the next advances in treatment development and ultimately a cure.

FRAXA shares Shionogi’s update on zatolmilast Fragile X clinical trial findings, what they mean for families, and details on the upcoming webinar.