Support Breakthroughs in Fragile X Research
For 22 years, Jason Mazzola’s life was shaped by Fragile X syndrome. Severe anxiety, communication challenges, and constant supervision defined his world.
Then, in May 2023, everything changed for Jason when he completed a double-blind, placebo-controlled clinical trial and was able to continue taking zatolmilast as part of an open-label extension. This promising drug, still in trials, has the potential to transform life for people with Fragile X.
Today, Jason walks downtown by himself, gets his haircut on his own, and even holds a job. His mother, Lizzie, reflects on the incredible change:
“He wouldn’t have done any of this before.”
This breakthrough, which was recently featured on NPR, brings hope not just to Jason but to countless others living with Fragile X. Families are seeing changes they once thought impossible: significant improvements in memory, language, and overall cognitive function, offering the hope of a better, more independent future.
Why Your Support Matters More Than Ever
“I have a different child in my house.”
– Lizzie Mazzola, Jason’s mother
This kind of progress — making the impossible possible — is only achievable with the support of people like you.
Your support of FRAXA-funded research is bringing us closer to the first FDA-approved treatment for Fragile X. We won’t know for sure until the current double-blind trials of zatolmilast are completed and analyzed, hopefully sometime next year.
But that’s not all. No single drug will work for everyone. One approved drug for Fragile X would be just the beginning. We are already working with teams to develop treatments with even more potential.
The efforts going on behind the scenes are simply amazing.
With your help these efforts will deliver results.
Paving the Way for New Fragile X Treatments
FRAXA is funding cutting-edge research in Antisense Oligonucleotide (ASO) therapy, a treatment that addresses the root cause of Fragile X rather than just the symptoms. With your support, ASO therapy could reach clinical trials within a few years, providing a revolutionary new option for families.
At the same time, FRAXA-funded researchers are developing gene reactivation and protein replacement therapies. These approaches have the potential to correct the underlying cause of Fragile X, but both involve major technical obstacles which have been very difficult to overcome. Only through many years of FRAXA-funded research have we been able to understand enough about the Fragile X mutation to make these therapeutic strategies feasible.
Those investments are close to paying off!
Collaborating for a Cure
FRAXA is in constant discussions with teams — academic and industry — brainstorming, planning and building the next steps toward effective treatments and ultimately a cure for Fragile X. These are extraordinarily complex endeavors; nothing is more complicated or precious than the human brain.
With your gift FRAXA can continue to fund life-changing studies, support essential clinical trials, and ensure families receive the groundbreaking treatments they need.
You can turn hope into reality for families affected by Fragile X.
