New hope for kids with rare epilepsy: experimental drug targets root cause

NCT ID NCT07363603

First seen Jan 23, 2026 · Last updated May 15, 2026 · Updated 20 times

Summary

This study tests an experimental drug called ASO-GNAO1 (Tianasen) in 5 children aged 1-14 with a specific genetic form of epilepsy and movement disorders. The drug is given via spinal injection over 12 months to see if it can reduce seizures and improve symptoms. Because the disease is rare and severe, all participants receive the drug, and researchers closely monitor safety and effectiveness.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

Study contacts

  • Contact

    Phone: •••-•••-•••• Email: •••••@•••••

Locations

  • Veltischev Research and Clinical Institute for Pediatrics and Pediatric Surgery of the Pirogov Russian National Research Medical University

    RECRUITING

    Moscow, Russia

    Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.