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Updated: Mar 7, 2026

Modeling Charcot-Marie-Tooth Disease In Vitro by Transfecting Mouse Primary Motoneurons
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An open-label single-arm phase 1/2a study to evaluate the safety and exploratory efficacy of a VM202 in patients with

Hye Mi Kwon1, Hyun Su Kim2, Sang Ah Chi3,4

  • 1Department of Precision Medicine, Sungkyunkwan University School of Medicine, Suwon, Republic of Korea.

Orphanet Journal of Rare Diseases
|March 5, 2026
PubMed
Abstract

No abstract available in PubMed .

Keywords:
Charcot-marie-tooth diseaseGene therapyHepatocyte growth factorPMP22

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