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ChinaResearch and clinical trials

Metformin trial in fragile X syndrome did not meet its main goal

Peer-reviewed publication

A study published in a journal and reviewed by independent scientists. Even so, a single study is not conclusive; it needs to be replicated.

In China, a six-month drug trial was carried out with 34 children with fragile X syndrome. Some children received metformin and some received an inactive drug. There was no significant difference between the two groups in the main goal, a behavior score. However, improvement was seen in hyperactivity and sleep problems. Side effects were similar in both groups.

What does it mean for patients and families?

This is a small study, and the drug is not approved for this disease. The results should be confirmed by new studies. Drugs should not be started without consulting a doctor.

Source

PubMed (Molecular Autism): Effects of Metformin on children with Fragile X Syndrome: a randomized, double-blind, placebo-controlled trial.

Scientific paper: Effects of Metformin on children with Fragile X Syndrome: a randomized, double-blind, placebo-controlled trial. Molecular Autism, 2025. DOI: 10.1186/s13229-025-00691-z · PubMed 41291951

The source page was opened and read on October 7, 2026. Summary prepared by the Doktorclub editorial team and translated from Turkish; it has not yet been reviewed by a physician (a scientific board is being set up).

This page is translated from the Turkish original.

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This page was translated from Turkish by the Doktorclub editorial team.