Fragile X Treatment Target Emerges from Neurolixis & FRAXA Collaboration

June 24, 2024

FRAXA pharma partner Neurolixis has filed patents for treatment of Fragile X with a highly selective new drug, NLX-101, which targets the serotonin receptor 5-HT1A.

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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.

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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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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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Correcting the Brain’s Emotional Memory Center

October 19, 2021

FRAXA Investigator Dr. Sumatra Chattarji investigated the synaptic basis of deficient conditioned fear and its reversal in Fragile X syndrome rats. Results published.

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Purposeful and FRAXA Partnership Leads to Clinical Trial

October 19, 2021

AI and FRAXA-DVI identified a drug + supplement combo that reversed all Fragile X symptoms in mice. A clinical trial tested this in adults with Fragile X.

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Aripiprazole (Abilify) in the Treatment of People with Fragile X: An Anecdotal Account

July 29, 2020

The aim of this article is to discuss the use of Abilify (generic name: aripiprazole) as a treatment for people with Fragile X syndrome (FXS). As an “off-label” prescription, Abilify targets behaviors such as irritability, aggression, self-injury and severe tantrums.

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Considering Available Drugs for Fragile X: My Favorite Combination (So Far)

February 19, 2020

Which of the available drugs are best for managing fragile X syndrome? Most drugs have “off-target” effects which can have key advantages in some cases.

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Meng Li, PhD, Xinyu Zhao, PhD, and Anita Bhattacharyya, PhD

FRAXA Research Grants Drive Big Investments in Fragile X

March 28, 2018

Most people know that FRAXA supports academic research at many institutions such as Harvard University, University of Pennsylvania, Massachusetts Institute of Technology, and Yale University. However, FRAXA is also working with more than 30 pharmaceutical companies around the world. Mike spends a lot of his time advising and collaborating with industry partners.

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Quantitative Assessment of the Serotonin System in a Mouse Model of Fragile X Syndrome

September 27, 2017

FRAXA funded Dr. Canal to investigate how different serotonin receptors function in Fragile X, to guide smarter use of serotonin-targeting treatments.

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Clinton Canal, PhD

Targeting Serotonin Receptors to Treat Behavioral and Psychological Symptoms

August 1, 2017

With a $90,000 grant from FRAXA Research Foundation awarded in 2017, Dr. Clinton Canal targets seratonin receptors. “There are 15 unique serotonin receptors (at least) and many of them impact the function of brain circuits that are impaired in neurodevelopmental and psychiatric disorders,” said Dr. Canal. “Results from this project could guide new drug discovery or drug repurposing for Fragile X.”

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Preclinical Evaluation of Serotonin Receptor Agonists as Novel Pharmacological Tools in Fragile X Syndrome

February 22, 2013

With FRAXA funding the team found that activating 5-HT7 receptors reversed excess mGluR-LTD in Fragile X mice, pointing to a new route to fix synapses.

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Serotonergic Rescue of Synaptic Plasticity in FMR1 Knockout Mice

January 31, 2012

Dr. Zhu examined how serotonin-targeting drugs such as Buspar and Abilify influence synaptic plasticity, including LTP and LTD.

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Social Deficits in Fragile X Syndrome: Do Gene-Gene Interactions Play a Role?

February 13, 2006

FRAXA’s $100K grant supported Drs. Jean Lauder and Sheryl Moy at UNC in exploring gene–gene interactions in Fragile X.

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Melatonin Clinical Trial in Fragile X

February 27, 2000

FRAXA-funded Dr. Hagerman’s UC team explored melatonin’s effects in Fragile X, advancing understanding of treatment options. Results published.

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FRAXA Funded Research

Current Research Grants (38)