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.

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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…

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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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.

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Charity Golf Tournament on Memorial Day Weekend Helps Fund Fragile X Research

June 29, 2022

Brothers Pete & Dave Hall’s 33rd Annual Golf Tournament raised $28K for FRAXA, bringing their 15-year total to over $262K!

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Transcriptional Signatures Sensitive to Cognition-Improving Pharmacological Treatments in Fragile X Syndrome

June 27, 2022

This team is defining Fragile X “molecular signatures” to use as biomarkers. They’ll test CBD and other drugs in mice and compare findings to human brain data.

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Built Jobsite BBQ Raising Funds for Fragile X Research

May 5, 2022

Built employee Jason and his team hosted a BBQ fundraiser for FRAXA after his sons, Jaxson and Alex, were diagnosed with Fragile X.

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Repurposing FDA-Approved Drugs to Treat Major Depressive Disorder in Fragile X Syndrome

April 24, 2022

Depression is common in Fragile X, but current antidepressants need FMRP to work. Researchers will screen FDA-approved drugs to find effective options for FXS.

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FMR1 Renamed to Fragile X Messenger Ribonucleoprotein 1

April 13, 2022

The FMR1 gene is renamed to “Fragile X Messenger Ribonucleoprotein 1” gene. The Fragile X protein, FMRP, is "Fragile X Messenger Ribonucleoprotein." "Mental retardation" is not used.

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VLM Commodities Charity Week Raises $10,000 for Fragile X Research

April 8, 2022

“Living with Fragile X is what we do—we keep going forward,” Lou shared. “FRAXA gives purpose, and I’m proud to support Charity Week.”

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How FRAXA Prioritizes Research, Explained

April 7, 2022

Dr. Mike Tranfaglia explains how FRAXA prioritizes research. "It’s not either-or. It’s not we have a definitive treatment or we have a new drug treatment or we have a repurposing treatment.

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Cape Coral Touch-A-Truck, Free, Family-Friendly Event

March 31, 2022

Join EHL Fragile X Foundation’s free Touch-A-Truck in Cape Coral! Explore fire trucks, police bikes, ambulances, swamp buggies, and more.

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Wine and Friends Are a Great Blend for Fragile X Research

March 25, 2022

Debra and Kevin hosted their first annual event for FRAXA in honor of their grandson Jackson, who has Fragile X syndrome.

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What FRAXA Is Excited about in the Upcoming Fragile X Research Grants, Explained

March 24, 2022

Many new clinical trials are starting, with existing and investigational drugs and other treatment strategies that have been tested in the Fragile X mouse models moving to patients in clinical trials.

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Gordon Research Conference for Fragile X and Autism-Related Disorders, Explained

March 17, 2022

The Fragile X and Autism-Related Disorders Gordon Research Conference is a biannual event, an international forum for the presentation and discussion of frontier research in these conditions.

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Making Mittens for Fragile X

February 25, 2022

Carol spends over 2 hours crafting each fleece-lined mitten. Her handmade creations warm hands and support Fragile X research.

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Meet Archie!

February 18, 2022

Archie really loves fire engines and trains as well as bells. Archie has had a few struggles this past year as his mom was diagnosed with cancer but thankfully she’s now cancer free.

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Banerjee team at College of Staten Island

Correcting Fragile X Syndrome Deficits by Targeting Neonatal PKCε Signaling in the Brain

February 17, 2022

Enhancing PKCε in early development normalized oxytocin, AMPAR signaling, and adult behavior in Fragile X mice, highlighting PKCε as a promising therapeutic target.

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FRAXA Funded Research

Current Research Grants (41)