News & Updates • Page 9 of 27 • FRAXA Research Foundation

Meet Chloe!

September 17, 2021

Meet #FriendofFRAXA Chloe! She is a bright girl who loves to make a joke and see people smile! If you would like to nominate someone as a #FriendofFRAXA, we welcome all who live with Fragile X.

Contribution of Microglia to the Therapeutic Effects of Metformin and Adiponectin in Fragile X Syndrome

September 16, 2021

Why are some with Fragile X always hungry or overweight, yet rarely diabetic? This team is studying metabolism and testing treatments like metformin and diet.

20 Years of Advancing Fragile X Research: Progress Toward a Cure

September 13, 2021

MIT Professor Mark Bear traces the discoveries that give us great optimism of finding effective treatments and ultimately a cure for Fragile X syndrome.

Meet Noah!

September 10, 2021

Meet #FriendofFRAXA Noah! Noah loves learning, playing outside, and riding in Mimi’s car. He’s really excited about starting public school.

Brain Organoids, Moving Fragile X Research Forward

September 9, 2021

FRAXA-funded scientists at Emory created human brain organoids that reveal Fragile X changes more clearly than mouse models, opening new paths to targeted treatments.

Tetra’s Fragile X Clinical Trial – The Most Successful So Far

September 7, 2021

Dr. Mark Gurney of Tetra Therapeutics, discusses how one of the earliest clues to the biology of Fragile X led to the most successful Fragile X clinical trial to date: a phase 2 trial of a PDE4D inhibitor.

Alternative Splicing in White Blood Cells: A Biomarker for Fragile X Syndrome

September 2, 2021

This team found 1,600 blood-based Fragile X biomarkers that vary by individual—opening the door to personalized treatment and better ways to measure progress.

Promising Results of Preclinical Study of ANAVEX®2-73

August 26, 2021

Anavex Life Sciences announced today that preclinical data of the ANAVEX®2-73 (blarcamesine) study in Fragile X syndrome were published in the journal, Scientific Reports.

Raising Awareness and Funds for Fragile X Research at Barre3

August 23, 2021

“A fun and sweaty way to unite the community!” FRAXA thanks Katie Bolin and Barre3’s Katie Heaps for supporting Fragile X awareness.

Fragile X Syndrome and Air Travel

August 19, 2021

If you wish to participate in a letter writing campaign you can copy the letter below and start sending it the CEOs listed at the bottom of the page.

Meet Jacob!

August 13, 2021

Meet Jacob! Jacob has a heart for helping others. He loves to serve and support those around him. He is kind, caring, and empathetic, and he always enjoys a good joke.

Synaptogenix Announced Intention to Launch a Fragile X Clinical Trial with Bryostatin

August 6, 2021

Bryostatin research has advanced from mouse models to human trials. Synaptogenix and Nemours make plans to test this potential treatment in Fragile X clinical trials.

Meet Benjamin!

July 30, 2021

Meet #FriendofFRAXA Benjamin! His smile lights up the room. He is a burst of energy! He gives the best hugs and loves to cuddle. You are welcome to nominate someone as a #FriendofFRAXA.

Fragile X Syndrome: In Pursuit of a Cure Webinar

July 29, 2021

Global webinar “Fragile X Syndrome: In Pursuit of a Cure” on July 22, 2021 commemorated World Fragile X Day. Over 5,000 registered from more than 50 countries.

2021 FRAXA Awards – Recognizing Perseverance and Dedication

July 22, 2021

In conjunction with World Fragile X Day 2021, FRAXA Research Foundation recognizes its annual award recipients. We are fortunate to partner with these individuals for Fragile X families.

Wieber Family Journey

July 22, 2021

In 2012, Zach and Leslie Wieber learned all three of their sons have Fragile X syndrome it was a moment filled with both relief and fear.

Link Between Lipid Profile, eCBome System and Gut Microbiome in Fragile X Syndrome

July 15, 2021

Why does obesity challenge so many people with Fragile X? Dr. Caku’s team has found that Fragile X syndrome causes changes in the tiny organisms that live in our gut.

USA Today, “Fragile X treatment: Decades later, progress in rare genetic condition”

July 12, 2021

USA Today profiled FRAXA co-founders Mike Tranfaglia, Katie Clapp, and their son Andy, highlighting how science is transforming rare disease care.

USA Today Video, “Living with Fragile X Syndrome: ‘He is growing… it’s just really slow'”

July 11, 2021

Born with Fragile X Syndrome, a rare developmental disorder, Andy Tranfaglia, thrives with the help of his parents and a community of supporters.

Characterization of Microglia Transcriptional Profile in Fmr1 Knockout Mice Model

July 1, 2021

Microglia are excessively activated in Fragile X models. The team will investigate the mechanisms and attempt to correct this using drugs.

The Role of Astrocyte BMP Signaling in Fragile X Syndrome

June 17, 2021

Researchers found a pathway in astrocytes that is overactive in Fragile X syndrome, and they hope to bring this pathway back to normal with a drug.

Drs. Oostra, Warren, and Nelson discovered the Fragile X gene and its FRAXA mutation in 1991.

Memorial Tribute to Dr. Stephen T. Warren

June 8, 2021

Dr. Stephen T. Warren, who discovered the Fragile X gene (FRAXA) in 1991, passed away June 6, 2021. Donations in his honor support FRAXA Research.

Meet Tyler!

June 7, 2021

Meet #FriendofFRAXA Tyler! Tyler enjoys visiting cemeteries to find his ancestors and loved ones. He also loves baking, listening to music, and going to train shows.

Identifying Cellular and Molecular Signatures in Human Neurons That Distinguish Fragile X Syndrome Patients with Divergent EEG Profiles

June 4, 2021

Just as Fragile X affects individuals differently, medications do as well. This project aims to bring personalized medicine to Fragile X syndrome.

Meet Chloe!

Contribution of Microglia to the Therapeutic Effects of Metformin and Adiponectin in Fragile X Syndrome

20 Years of Advancing Fragile X Research: Progress Toward a Cure

Meet Noah!

Brain Organoids, Moving Fragile X Research Forward

Tetra’s Fragile X Clinical Trial – The Most Successful So Far

Alternative Splicing in White Blood Cells: A Biomarker for Fragile X Syndrome

Promising Results of Preclinical Study of ANAVEX®2-73

Raising Awareness and Funds for Fragile X Research at Barre3

Fragile X Syndrome and Air Travel

Meet Jacob!

Synaptogenix Announced Intention to Launch a Fragile X Clinical Trial with Bryostatin

Meet Benjamin!

Fragile X Syndrome: In Pursuit of a Cure Webinar

2021 FRAXA Awards – Recognizing Perseverance and Dedication

Wieber Family Journey

Link Between Lipid Profile, eCBome System and Gut Microbiome in Fragile X Syndrome

USA Today, “Fragile X treatment: Decades later, progress in rare genetic condition”

USA Today Video, “Living with Fragile X Syndrome: ‘He is growing… it’s just really slow'”

Characterization of Microglia Transcriptional Profile in Fmr1 Knockout Mice Model

The Role of Astrocyte BMP Signaling in Fragile X Syndrome

Memorial Tribute to Dr. Stephen T. Warren

Meet Tyler!

Identifying Cellular and Molecular Signatures in Human Neurons That Distinguish Fragile X Syndrome Patients with Divergent EEG Profiles

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