First human test of new cell therapy for inherited nerve disease

NCT ID NCT06328712

Summary

This was a very early, small study to check the safety of a new cell therapy called EN001 for people with Charcot-Marie-Tooth disease type 1A (CMT1A), a genetic nerve disorder. Six adult participants received two doses of EN001 to find the highest safe amount. Researchers monitored them closely for side effects and also looked for any early signs that the treatment might help with symptoms like muscle weakness.

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Contacts and locations

Locations

  • Samsung Medical Center

    Seoul, South Korea

Conditions

Explore the condition pages connected to this study.