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🎉 Congratulations to FRAXA Investigator Dr. Lynne Maquat, recipient of the 2024 Dr. Paul Janssen Award! 🏆 Her research on nonsense-mediated mRNA decay (NMD) has been crucial in understanding diseases like cancer and #FragileX. urmc.rochester.edu/news/story/lyn…

The latest advances in #FragileX research are bringing us closer to new treatments. Kaerus Bioscience & FRAXA are developing BK channel openers to address core challenges in Fragile X. We’re moving toward transformative treatments. fraxa.org/bk-channel-ope… #GeneTherapy #ClinicalTrials

We’re launching our 2024 Annual Appeal with a $100K gift from the Berg & Schatz families in honor of young Cooper, who has #FragileX. Their generosity will fund 2 research teams, advancing us toward treatments & a cure. Every donation matters. #FXResearch fraxa.org/kicking-off-ou…

For 22 yrs, Jason’s life was shaped by #FragileX. In 2023, thanks to a #clinicaltrial & its open label extension, everything changed. Jason now walks downtown, gets his haircut, and holds a job. “He wouldn’t have done any of this before,” says his mom. fraxa.org/annual-appeal-…

This Thanksgiving, we’re grateful for your support of FRAXA’s mission to find effective treatments and a cure for #FragileX. Together, we’ve driven progress, supported families, and inspired #hope.

The #FragileX journey is challenging, but you don’t have to navigate it alone. There’s still time to join us on December 12 at 12pm ET for Fragile X Family Conversations: Sharing, Learning, Supporting – a virtual gathering for families and caregivers. fraxa.org/events/fragile…

We’re investigating potential treatments for symptoms associated with rare neurobehavioral disorders, including #FragileX syndrome. Learn more: bit.ly/3TSDDOK #FXS #FragileXawareness

FRAXA is launching a bold new grant program to accelerate curative therapies for #FragileX, including gene therapy, protein replacement, & mRNA therapeutics. Researchers can apply for up to $100k/yr. Let’s drive progress toward life-changing treatments! fraxa.org/fragile-x-rese…

🧬 Join us Feb 11 at 12:00 pm ET for #FragileX Family Conversations—a supportive space to connect, share, and support each other. As parents ourselves, FRAXA offers a space where families feel seen, heard, and hopeful. Seats are limited—register now! fraxa.org/events/fragile…

The RECONNECT #clinicaltrial is enrolling individuals with #FragileX between the ages of 3-29. This study is evaluating an investigational treatment for behavioral symptoms associated with FXS. With both at-home and in-clinic options, Find out more: fraxa.org/recruiting-har…

#Clinicaltrials help advance #research toward new treatment options. The RECONNECT study by Harmony Biosciences is evaluating an investigational treatment for #FragileX & is currently enrolling. Families can choose to participate from home or visit a study site. fraxa.org/recruiting-har…

FRAXA-funded researchers at Stanford are studying ISRIB, an experimental drug that may help restore brain function in #FragileX. >Improves brain connectivity >Strengthens neural communication >Enhances social behavior in FX models fraxa.org/fraxa-funded-r…