Urgent Action Needed: Help Secure NIH Funding for Fragile X Research

NIH funding delays threaten Fragile X research, putting critical studies and future treatments at risk. Help us urge Congress and the NIH to act now.

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Renewed Hope: Navigating Towards a Cure for Fragile X Syndrome

Discover how Dr. Peter Todd’s latest Fragile X Syndrome research offers hope for advanced treatments and a possible cure, marking a new era in FXS therapy.

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ASOs and Fragile X: Addressing the Most Asked Questions

Explore the potential of ASOs in treating Fragile X syndrome & FXTAS. Dive into a comprehensive Q&A addressing key questions and breakthrough findings.

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2023 Fragile X Research Banbury Meeting (1)

Breakthrough Discoveries in Fragile X Research: Insights from Special Banbury Meeting on Curative Therapies

Explore the latest breakthroughs in Fragile X research unveiled at the recent Banbury Meeting. Discover novel strategies, from gene therapy to protein replacement, that bring hope for curative therapies.

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

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.

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Towards Understanding the Role of FMRP in Human Brain Development Using Brain Organoids

Dr. Zhexing Wen and Dr. Peng Jin at Emory University School of Medicine join us in this seminar to present Understanding the Role of FMRP in Human Brain Development Using Brain Organoids.

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Elle Skala, Tracy King, Christie Rogers, Mary Beth Busby

National Institutes of Health Releases Fragile X Strategic Plan

FRAXA’s Elle Skala and supporter Mary Beth Busby visited NIH as the new Strategic Plan for Fragile X was released, shaping federal research priorities for years.

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How Promising is CRISPR for Fragile X?

Peter Todd, MD, PhD, Assistant Professor in the Department of Neurology in the University of Michigan Medical School, was awarded a FRAXA Research Grant for gene reactivation with the use of CRISPR. In this interview he tells us about CRISPR in Fragile X research, how realistic is it that it could turn the Fragile X gene back on, and if it can really cure Fragile X.

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David Nelson, PhD, FRAXA Investigator

Fragile X Mutant Mouse Models

With $375,000 in grants from FRAXA, Dr. David Nelson developed an array of advanced mouse models of Fragile X. These models are available at Jackson Labs (JAX).

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Dr. Haenfler and Dr. Todd

University of Michigan researcher Peter Todd, MD, PhD, Aims to Selectively Turn the Fragile X Gene Back on in Human Cells

University of Michigan scientists Peter Todd, MD, PhD, and postdoctoral fellow Jill Haenfler, Ph.D., are adapting CRISPR to reactivate the FMR1 gene to reverse Fragile X syndrome.

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David Nelson lab

Researcher David Nelson, PhD, Explores New Cell Strategies for Fragile X Syndrome, FXTAS and FXPOI

David Nelson co-discovered the FMR1 mutation which causes Fragile X syndrome and developed key mouse models, enabling global research aimed at finding treatments for the disorder.

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Kimberly Huber, Ph.D., FRAXA Investigator

NIH Awards $35 Million to Three Fragile X Research Teams

NIH is investing $35M in three Fragile X Research Centers. All teams have been funded by FRAXA and will now receive over $2M annually for five years.

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What Treatments Work for FXTAS?

FXTAS affects many in our Fragile X community. Research aims to uncover its cause and guide more effective treatments.

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Small Molecules To Target r(CGG) Expansions to Treat Fragile X Syndrome

FRAXA-funded scientists created small molecules that target the CGG repeat “off-switch” in Fragile X, aiming to restore the missing FMRP protein at its source.

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Efficient Screening for Pharmaceutical Amelioration of FXS Behavioral Deficits in Drosophila

Using a fruit-fly Fragile X model, researchers screened many drugs quickly to find those that improve behavior, speeding up potential treatment testing.

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FRAXA Funded Research

Current Research Grants (37)