FRAXA-Funded Research Explores ISRIB as a Potential Treatment for Fragile X

ISRIB for Fragile X syndrome is being studied as a potential treatment to restore brain function and social behavior. Researchers investigate its effects.

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Two-Med Combo Normalized Behavior, Improved Memory in Fragile X Mice

Treating Fragile X might require a combination of drugs. FRAXA-DVI tested two investigational drugs in Fragile X mice. Together ibudilast and gaboxadol rescued a wide array of symptoms.

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Versatile Drug Screening Platform for Fragile X Syndrome

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.

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New Fragile X Clinical Trial Announced by Healx

Healx’s AI-driven approach makes finding the right combination therapies more efficient, cost-effective, and rapidly ready for testing at FRAXA-DVI. It was this process that has brought Healx to its recent announcement sharing that it has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for the Phase 2a clinical study of HLX-0201 (sulindac, an FDA-approved drug).

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Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome

Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome

This Stanford University team assessed combinatorial drug treatments to correct a broad spectrum of deficits observed in Fragile X syndrome. Results published.

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Healx team 2018

Repurposing Study II: Evaluating Combinations of Drugs to Treat Fragile X

FRAXA partnered with Healx to use AI to find approved drugs and drug combos that could treat Fragile X. Top candidates are now being tested in Fragile X models.

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Funding opportunities - FRAXA investigators

Combinatorial Drug Treatment in a Model of Fragile X Syndrome using Novel Biomarkers

University of California researchers Khaleel Razak, PhD, and Jonathan W. Lovelace, PhD, explored drug combinations to limit hypersensitivity to sounds in Fragile X mice.  

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Dr. Jonny Lovelace and Dr. Khaleel Razak

Meltdown no more? Targeting Hypersensitivity in Fragile X

Meet Khaleel Razak, PhD, and Jonathan W. Lovelace, PhD, FRAXA-funded researchers at University of California, Riverside who are tackling Fragile X.

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Andy and MIke Tranfaglia

Why Did Fragile X Clinical Trials of mGluR Antagonists Fail?

by Michael Tranfaglia, MD. In my opinion, the Fragile X clinical trials of AFQ056 sponsored by Novartis failed because of a dose range that was inadequate for Fragile X, and because of the unexpected development of tolerance.

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Baclofen: GABA(B) Receptor Supersensitivity and Normalization of Behavioral Abnormalities by Various GABA(B) Agonists Including Baclofen in FMRP Deficient Mice

FRAXA’s $110K grant supported Dr. Miklos Toth at Cornell in discovering that baclofen corrects the heightened startle response in Fragile X mice.

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FRAXA Funded Research

Current Research Grants (38)