New drug shows promise for rare muscle disease in early trial

NCT ID NCT04004000

First seen Jan 09, 2026 · Last updated May 15, 2026 · Updated 18 times

Summary

This study tested a drug called losmapimod in 14 adults with facioscapulohumeral muscular dystrophy type 1 (FSHD1), a rare genetic condition that causes muscle weakness. The main goal was to check if the drug is safe and tolerable, and to see how it affects certain markers in the blood and muscles. The trial was stopped early, but the results help researchers understand if losmapimod might control the disease.

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Contacts and locations

Locations

  • Radboud University Medical Center

    Nijmegen, 9101, Netherlands

Conditions

Explore the condition pages connected to this study.