Gene therapy for krabbe disease: researchers track Long-Term safety and progress

NCT ID NCT06308718

First seen Nov 01, 2025 · Last updated May 13, 2026 · Updated 30 times

Summary

This study follows up with Krabbe disease patients who previously received FBX-101 gene therapy in earlier clinical trials. The goal is to check for long-term side effects and see how well their motor skills are maintained over time. Only 2 participants were enrolled, and no new treatments are given—just observation and monitoring.

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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

Locations

  • University of Michigan Hospitals - Michigan Medicine

    Ann Arbor, Michigan, 48109, United States

Conditions

Explore the condition pages connected to this study.