drug combinations Archives

Nicola Elvassore and his research team at the University of Padua, Italy, in their laboratory conducting Fragile X brain organoid studies supported by FRAXA Research Foundation

Cell Type-Specific Protein Dysregulation in Fragile X Syndrome Brain Organoids

November 10, 2025

Study finds that brain cell types respond differently to gene silencing, pointing to the need for customized treatment approaches in Fragile X.

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

February 26, 2025

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

Screening Combinatorial Pharmacological Therapies for Fragile X Syndrome

October 9, 2024

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

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

March 6, 2024

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

Versatile Drug Screening Platform for Fragile X Syndrome

November 2, 2022

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.

New Fragile X Clinical Trial Announced by Healx

October 21, 2021

Healx has received Investigational New Drug (IND) approval from the US Food and Drug Administration (FDA) for a Phase 2a clinical study of HLX-0201 (sulindac, an FDA-approved drug).

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

February 8, 2018

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.

Funding opportunities - FRAXA investigators

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

October 3, 2017

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

Dr. Jonny Lovelace and Dr. Khaleel Razak

Meltdown no more? Targeting Hypersensitivity in Fragile X

June 5, 2016

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

Why Did Fragile X Clinical Trials of mGluR Antagonists Fail?

August 15, 2014

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.