Gene shot tested for rare nerve disease
NCT ID NCT05361031
Trial info
This early-stage study tested the safety of a new gene-based injection called Engensis in people with Charcot-Marie-Tooth disease type 1A (CMT1A), a genetic nerve disorder. Twelve adult participants received injections into their weakened leg muscles over 270 days. The main goal was to see if the treatment was safe, while also checking for any signs it might help with walking, muscle strength, and nerve function.
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Contacts and locations
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Locations
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Samsung Medical Center, Seoul National University Hospital
Seoul, 06351, South Korea
Conditions
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