Dr. Thomas Maurin and Dr. Barbara Bardoni, Fragile X researchers and co-authors of a 2025 review on PDE inhibitors published in Cell Reports Medicine.

Research Points to Drugs which Inhibit PDE to Treat Fragile X

FRAXA-funded work identified PDE enzymes as key targets in Fragile X, showing that PDE inhibitors can fix signaling and boost synaptic function. PDE4D trials are underway.

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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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Encouraging Results from First Trial of Minocycline in Fragile X

A clinical trial of minocycline in children with Fragile X found significantly better global improvement vs. placebo, supporting its safety and potential.

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Iryna Ethell, PhD, at University of California

Role of Matrix Metalloproteinases (MMP-9) in Fragile X

With a $220,000 FRAXA grant, Dr. Iryna Ethell’s team at UC Riverside uncovered MMP-9’s role in Fragile X—leading to a major treatment strategy using minocycline.

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FRAXA Funded Research

Current Research Grants (43)