News & Updates • Page 6 of 26 • FRAXA Research Foundation

Targeting Serotonin 1a Receptors to Reverse Neurobehavioral Phenotypes

January 14, 2023

Neurolixis’ new drug targets serotonin 1A receptors, showing promise in preclinical studies for Fragile X syndrome, funded by a FRAXA grant for future clinical trials.

Meet Ryder!

January 6, 2023

Meet #FriendofFRAXA Ryder! If you would like to nominate someone as a #FriendofFRAXA, we welcome all who have been touched by Fragile X. Ryder is a delight!

10 Year Vision for Fragile X Research – Dr. Elizabeth Berry-Kravis & Dr. Patricia Cogram

December 22, 2022

FRAXA Investigators Dr. Patricia Cogram and Dr. Elizabeth Berry-Kravis reflect on progress that has been made and visualize what they see happening in the next 10 years for people living with Fragile X syndrome.

mRNA Therapy for Fragile X Syndrome

December 11, 2022

Dr. Kathryn Whitehead helped develop the science behind the COVID-19 vaccines. Her team adapted this technology to deliver the Fragile X mRNA to brain cells.

$269,774 Raised for Fragile X Research on #GivingTuesday

December 2, 2022

Thank you for making #GivingTuesday historic! With matching gifts, FRAXA raised $269,774—an incredible show of support!

A Note of Thanks and Hope on This Giving Tuesday

November 29, 2022

Fragile X research matters. Each discovery brings us closer to treatments for autism and other brain disorders. Thanks to you, we have hope.

Clinical Trial Results Reported for Phase 3 CONNECT-FX Study of Zygel™

November 29, 2022

Disappointing results have been published from Zynerba Pharmaceuticals’s phase 3 clinical trial of Zygel™. 212 children and adolescents 3 to 17 years were given Zygel or placebo for 12 weeks.

Sigma-1 Receptor as a Therapeutic Target for Fragile X Syndrome

November 22, 2022

Researchers will test sigma-1 receptor drugs—like fluvoxamine, which activates this pathway—to see if they can correct Fragile X brain cell abnormalities.

Fragile X Clinical Trial of New PDE4D Inhibitor from Tetra

November 20, 2022

A $200K FRAXA grant enabled a successful Phase 2 trial of a PDE4D inhibitor for adult men with Fragile X, showing strong cognitive gains without side effects or tolerance.

$19,600 Raised For Fragile X Research at Callum Cup VI

November 7, 2022

The Callum Cup is back! This beloved Millburn FC charity match has raised $94,600 for Fragile X research over 7 amazing years.

Altered Sleep in Fragile X Syndrome: Basis for a Potential Therapeutic Target

November 5, 2022

With this FRAXA grant, Dr. Carolyn B. Smith and Dr. Rache Sare at the National Institute of Mental Health investigated the basis of sleep problems in Fragile X syndrome.

Versatile Drug Screening Platform for Fragile X Syndrome

November 2, 2022

Combinations of drugs may be needed to treat Fragile X syndrome. How can we find the best combinations in the ideal doses? This project tackles this challenge.

Human FMR1 Isoform-Specific Regulation of Translation and Behavior

October 19, 2022

FMRP has multiple forms, and this team will study which isoforms are most important for brain development. This is key for future FMRP replacement therapies.

Meet Conor!

October 14, 2022

Conor loves to swim and would stay in the pool all day if we let him. He loves taking the bus to school and a new "Boom Clap" game that they play. Challenges include…

Meet Tobias!

September 30, 2022

Tobias loves watching cooking shows and helping out in the kitchen. He really struggles with social anxiety and people he doesn’t know, also being the center of attention.

Functional and Genomic Characterization of Interneurons in the Fmr1-KO Mouse Brain

September 22, 2022

The brain’s balance is maintained by two types of neurons: excitatory and inhibitory. This team has found fewer than normal inhibitory cells in Fragile X mice.

Function of FMRP and Test of a Novel Therapeutic Approach in a Fragile X Mouse Model

September 16, 2022

FRAXA-supported work has identified DgkK as a critical enzyme lost in Fragile X. Drugs that raise DgkK levels may correct brain signaling and improve symptoms.

Targeting Serotonin 1A Receptors in Fmr1 Knockout Mice

August 30, 2022

Boosting serotonin 1A receptors may reduce excess brain activity in Fragile X. This study will test serotonin-1A–targeting compounds in mice to guide future treatments.

FRAXA Volunteer Participates in Peer Reviewed Medical Research Program for the Department of Defense

August 26, 2022

FRAXA advocate Jennifer Frobish served as a reviewer for the Department of Defense’s medical research program, evaluating Fragile X–related proposals.

Families Come from Multiple States for Flatbread Pizza Fundraiser

August 19, 2022

We met Katie and other Fragile X families who welcomed us with open arms—it’s a wonderful feeling to know we’re not alone.

Patrick’s PALS 3-on-3 Basketball Tournament Returns with a Big Impact

August 10, 2022

Patrick’s PALS 3-on-3 Basketball Tournament returned after 3 years, raising nearly $150K for FRAXA with help from Shady Hill School!

World Fragile X Day Is Lighting the Way for Fragile X Research

August 8, 2022

On July 22, 383 landmarks worldwide lit up for World Fragile X Day—shining bright to raise awareness and celebrate research progress.

Why Pharma Companies Take on Fragile X, Explained

June 30, 2022

Research aimed at finding Fragile X syndrome treatments is exploding. Why are so many companies investing? FRAXA CSO Dr. Michael Tranfaglia explains many reasons Fragile X is a hot topic.