Article

A phase 1/2a, open label study to evaluate the safety and efficacy of a plasmid DNA encoding human hepatocyte growth factor in patients with Charcot-Marie-Tooth disease 1A

2024-06-14

Abstract excerpt

<title>Abstract</title> <p> <bold>Background</bold> This is the first gene therapy trial in patients with Charcot-Marie-Tooth disease type 1A (CMT1A). Intramuscular injectionsof VM202, a plasmid DNA encoding human hepatocyte growth factor, was safe, tolerable and potentially effective in patients with CMT1A. <bold>Methods</bold> This study was a phase 1/2a, nonrandomized controlled trial. Thirteen patients wi...

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Literature Corpus work: fee6e42c-c373-530c-a796-95f33eb00c5f
DOI: 10.21203/rs.3.rs-4220356/v1

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