20-Year study reveals precision Medicine's promise for rare disorders

NCT ID NCT07327164

First seen Jan 11, 2026 · Last updated May 15, 2026 · Updated 17 times

Summary

This study followed 1,200 people with neurocutaneous syndromes (like NF1, TSC, and Sturge-Weber) over 20 years to see if genetic testing and targeted drugs improve survival, seizure control, and quality of life. Researchers compared those who received precision medicine (genetic testing + targeted drugs + coordinated care) to those who got standard care. The goal was to learn if these approaches work better and cost less in Western China.

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Contacts and locations

Locations

  • West China Hospital of Sichuan University

    Chengdu, Sichuan, China

Conditions

Explore the condition pages connected to this study.