Fragile X Syndrome Timeline

Fragile X protein is identified and cloned by the gene’s discoverers. This protein, FMRP, is lacking in people who have Fragile X syndrome.

FRAXA funds first research project led by Ted Brown, MD, PhD, New York State Institute for Basic Research, to develop a yeast model of Fragile X.

FRAXA begins first ever Fragile X advocacy in Washington, DC; Congress recognizes critical need for Fragile X research for first time.

Fragile X knockout mice shared with the worldwide research community. Study with these mice leads to first understanding of the cause of Fragile X.

Randi Hagerman, MD, receives FRAXA grant for clinical trial of melatonin to help Fragile X syndrome patients sleep.

While working on a FRAXA grant aimed at reactivating the FMR1 gene, Flora Tassone, PhD, and Paul Hagerman, PhD, made a groundbreaking discovery - the cause of Fragile X-Associated Tremor/Ataxia Syndrome (FXTAS) - increased FMR1 mRNA.

FRAXA organized and hosted the inaugural Fragile X Banbury Research meeting, which paved the way for many discoveries and trials. These meetings were held annually until 2011.

The US Senate established the first National Fragile X Awareness Day after the tireless efforts of FRAXA Research Foundation, David Busby (father of two sons with Fragile X syndrome), and the first Fragile X Senate Champions, John Edwards and Chuck Hagel.

Robert Darnell, MD, PhD, and Jennifer Darnell, PhD, made a groundbreaking discovery about Fragile X syndrome by identifying and publishing a list of the molecular targets of FMRP. Their research, made possible by a FRAXA funded research grant, led to a new understanding of the disorder.

FRAXA funds clinical trial of the available drug lithium by Elizabeth Berry-Kravis, MD, PhD to find out if it improves the behaviors and learning deficits seen in Fragile X syndrome. 

Thanks to the Doris Buffett Challenge, "The sky is the limit!" - an unlimited matching gift challenge - FRAXA Research Foundation was able to double its funding for Fragile X research.

FRAXA Drug Validation Initiative (FRAXA-DVI) funded to provide a standard platform to test potential treatments using mice. Goes on to complete preclinical testing of 100+ compounds.

First use of Artificial intelligence (AI) in Fragile X syndrome. FRAXA funds University of Cambridge (England) startup Healx to use Artificial Intelligence to identify the most promising available drugs to treat Fragile X syndrome.

The FRAXA-funded Phase 2 clinical trial conducted by Tetra Therapeutics investigating a PDE4 inhibitor reported significant cognitive improvement in Fragile X syndrome, marking the first unequivocally positive clinical trial result after years of rational drug discovery efforts.

FRAXA Research Foundation launches World Fragile X Day to celebrate individuals affected by Fragile X syndrome and their families, and to raise awareness worldwide.

FRAXA Research Foundation passes the $30 million mark in direct Fragile X research funding.

FRAXA Research Foundation hosts special Fragile X Banbury Research meeting to discuss breakthroughs in gene reactivation and gene therapy for Fragile X.