One patient, one drug: custom gene therapy trial launches for Ultra-Rare disease
NCT ID NCT07223632
Summary
This is a highly personalized study for a single patient with a rare genetic nerve disease called CMT2S. The trial is testing the safety of a custom-made drug, VCA-894A, designed to fix a specific genetic error. The drug is injected into the spinal fluid to try to restore a missing protein and control the disease's progression.
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Contacts and locations
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Locations
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Vanda Investigational Site
Madison, Wisconsin, 53792, United States
Conditions
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