The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)

Summary

To assess the safety and tolerability of the investigational product Engensis (VM202) injected in the weakened lower limb muscles of CMT1A patients

Timeline

Start

2020-09-21

Primary completion

2021-09-09

Completion

2022-02-22

Publications

• Kwon HM, Kim HS, Chi SA, Nam SH, Kim HJ, Kim SB, Choi BO. An open-label single-arm phase 1/2a study to evaluate the safety and exploratory efficacy of a VM202 in patients with Charcot-Marie-Tooth disease 1A. Orphanet J Rare Dis. 2026 Mar 5;21(1):148. doi: 10.1186/s13023-026-04252-2.

Drugs

Indications