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FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome

FRAXA Research Foundation – Finding a Cure for Fragile X Syndrome

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FRAXA Funded Research

Research Type

Researcher

Research Organization

Grant Year

Shah, Sneha

QurAlis and UMass Chan Advance Fragile X Syndrome Treatment using ASOs (Antisense Oligonucleotides)

FRAXA Research Foundation June 25, 2024June 25, 2024

Explore how QurAlis and UMass Chan are revolutionizing Fragile X syndrome treatment using advanced ASO technology, setting new standards in therapeutic development.

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Antisense Oligonucleotides (ASOs) to restore FMRP in Human Fragile X Cerebral Organoids

FRAXA Research Foundation August 24, 2023November 19, 2024

Explore Dr. Richter’s encouraging results with ASOs for Fragile X syndrome. A $100,000 grant now fuels pivotal studies needed to advance toward ASO therapy.

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Innovative Breakthrough in Fragile X Treatment: The Promise of Antisense Oligonucleotide (ASO) Therapy

Michael Tranfaglia, MD June 27, 2023August 22, 2023

This changes everything! FRAXA funded research introduces Antisense Oligonucleotide (ASO) Therapy, redefining Fragile X syndrome treatment and understanding.

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

FRAXA Research Foundation September 2, 2021March 3, 2025

Explore groundbreaking research by the University of Massachusetts Medical School and Rush University Medical Center on alternative splicing in white blood cells as a biomarker for Fragile X syndrome, paving the way for personalized treatment optimization through a non-invasive blood test.

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Deep Molecular Profiling of Fragile X Mouse and Human Cells

FRAXA Research Foundation September 5, 2019January 29, 2024
Deep Molecular Profiling of Fragile X

FRAXA Research Foundation has awarded $90,000 to Dr. Joel Richter, Principal Investigator, and Dr. Sneha Shah, Postdoctoral Fellow, at the University of Massachusetts Medical School. They are using human induced pluripotent stem (iPS) cells to analyze gene expression in Fragile X syndrome.

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