Clinical trials: Hereditary Motor and Sensory Neuropathy
CMT
The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)
To assess the safety and tolerability of the investigational product Engensis (VM202) injected in the weakened lower limb muscles of CMT1A patients
- Trial name
- NCT05361031
- Testing
- Engensis (VM202)
- Where
- South Korea
- Volunteers
- 12 people
- Sponsor
- Helixmith Co., Ltd.
Charcot-Marie-Tooth Disease With Sorbitol Dehydrogenase Deficiency (CMT-SORD)
Confirmatory Study of Govorestat in CMT-SORD
This study is designed to assess the long-term efficacy and clinical benefit of AT-007 in patients with CMT-SORD. This randomized, double-blind study will assess the effect of govorestat compared to placebo in patients with…
- Trial name
- NCT07191912
- Testing
- Govorestat
- Where
- 13 sites in United States, Australia and 5 more
- Sponsor
- Applied Therapeutics, Inc.