CMT is a disease of the peripheral nerves that control the muscles. It gradually damages motor neurons and sensory neurons in arms and legs, causing muscle weakness and walking disorders. CMT can be severely disabling in some people. CMT1A, the most common type of the disease, occurs due to PMP22 gene duplication and accounts for about 40% of all CMT patients, with approximately 20,000 patients in Korea and more than 3 million worldwide (CMT Association).
There is no FDA approved therapeutic medicine up to now and Phase 1/2a clinical trial for Engensis (vm202) is now in progress in Korea. By injecting Engensis (VM202) into the leg muscle of patients with muscular atrophy from damage of peripheral nerves, several activities such as regeneration of muscular tissue, inhibition of apoptosis, anti-inflammation, anti-fibrosis, and neuronal protection are induced.