30 Years of Outstanding Fragile X Research - The FRAXA Timeline
Discover the evolution of Fragile X research through the work of FRAXA Research Foundation, which is dedicated to finding effective treatments and ultimately a cure for Fragile X syndrome. This interactive timeline showcases milestones, breakthroughs, and key events that have shaped our evolving understanding of Fragile X syndrome.
Explore these pivotal moments to understand the unwavering dedication of researchers and FRAXA's far-reaching impact on countless lives. Join us as we look ahead to a future full of hope and promise for individuals and families affected by Fragile X.
1991
Scientists Discover the Cause of Fragile X
Ben Oostra, Stephen Warren, David Nelson, and others find a mutated gene on the X chromosome. They name the gene "FMR1" and the mutation site "FRAXA". This mutation prevents FMR1 from producing its normal protein, causing Fragile X syndrome.
1994
Parents Found FRAXA Research Foundation to Find Treatments and Ultimately a Cure
Katie Clapp, Michael Tranfaglia, and Kathy May establish FRAXA to drive research forward to improve the lives of families.
1995
FRAXA Funds First Gene Therapy Research
FRAXA funds the first research to adapt gene therapy for Fragile X, led by Jude Samulski at the University of North Carolina.
1995
FRAXA Goes to Washington, DC
FRAXA begins the first advocacy ever done in Washington, DC to accelerate Fragile X research and awareness. The U.S. Congress acknowledges the critical need for Fragile X research for first time.
1998
FRAXA Puts Fragile X on the Internet
FRAXA launches the first website dedicated to Fragile X. FRAXA also launches “the listserv” -- the first online Fragile X community was a global lifeline for many.
1998
FRAXA Funds Its First Clinical Trial
Randi Hagerman receives a FRAXA grant for a clinical trial to see if melatonin can help Fragile X syndrome patients sleep at the University of California at Davis.
1999
FRAXA Investigators Discover Cause of FXTAS
During their FRAXA-funded research Paul Hagerman and Flora Tassone make a surprise discovery: the cause of Fragile X-Associated Tremor/Ataxia Syndrome (FXTAS).
2000
Success in Washington!
The Children’s Health Act of 2000 launches Fragile X Research Centers of Excellence which still exist today. The US Senate establishes National Fragile X Awareness Day after tireless efforts of FRAXA advocates.
2005
FRAXA's Scientific Advisory Board Welcomes Three Nobel Laureates
FRAXA's Scientific Advisory Board expands to include three Nobel Prize winners. Two of these Nobel Laureates -- Eric Kandel and Susumu Tonegawa -- embark on research to cure Fragile X with grants from FRAXA.
2005
Big Pharma Joins the Race to Cure Fragile X
Roche and Novartis Pharmaceuticals both start investing millions to develop treatments for Fragile X after attending a Fragile X Banbury meeting hosted by FRAXA.
2010
New York Times Front Page Reports Successful Clinical Trial
The success of a clinical trial, featured on Bloomberg News, NPR, and the front page of the New York Times, marks a turning point for public awareness of Fragile X syndrome and the importance of continued research.
2011
FRAXA Launches Drug Validation Initiative in Chile
Funded continuously until today, FRAXA-DVI is a standard platform to test potential Fragile X treatments. It has completed preclinical testing of 100+ compounds. Successful candidates can move forward to clinical trials.
2016
First Use of Artificial Intelligence to Tackle Fragile X
FRAXA funds University of Cambridge (England) startup Healx to use Artificial Intelligence to identify the most promising available drugs to treat Fragile X syndrome.
2020
Success of Clinical Trial of PDE4D Inhibitor
A Phase 2 clinical trial funded by FRAXA and Tetra Therapeutics reports significant cognitive improvement in adults with Fragile X syndrome, marking the first unequivocally positive trial result after years of efforts.
2021
FRAXA Launches World Fragile X Day
FRAXA launches World Fragile X Day to honor individuals living with Fragile X and raise global awareness.
2022
FRAXA Launches Drug Testing Platform in Belgium
FRAXA funds a collaboration between Kantify, an Artificial Intelligence company, and the lab of Frank Kooy to build a new drug testing platform to evaluate treatments.
2023
FRAXA Investigator's Serendipitous Discovery Opens the Door to a Cure with ASO Therapy
While working on a FRAXA grant to pinpoint biomarkers, Joel Richter and colleagues at UMass Chan Medical School discovered a path to Fragile X treatment with antisense oligonucleotides (ASOs).
2024
Phase 3 Fragile X Clinical Trials are Underway
Pivotal clinical trials are accepting hundreds of participants for Tetra Therapeutics' PDE4D inhibitor and Zynerba Pharmaceuticals' CBD gel across the U.S. and in several other countries. First results are expected in 2025.
2024
Biotech Company Invests Millions to Develop ASO Treatment for Fragile X
QurAlis partners with UMass Chan Medical School to develop antisense oligonucleotide (ASO) treatment for Fragile X syndrome. The goal: a definitive treatment for patients of all ages.