CMT
Clinical trials for CMT explained in plain language.
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New Gene-Based injection tested for nerve disease safety
Disease control CompletedThis early-stage trial tested whether an experimental drug called Engensis (VM202) is safe when injected into the leg muscles of people with Charcot-Marie-Tooth disease type 1A (CMT1A), a genetic nerve disorder that causes muscle weakness. Twelve adults with mild to moderate CMT1…
Matched conditions: CMT
Phase: PHASE1, PHASE2 • Sponsor: Helixmith Co., Ltd. • Aim: Disease control
Last updated May 15, 2026 11:54 UTC
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New pill aims to help CMT patients walk farther in just 3 weeks
Symptom relief CompletedThis study tested an experimental drug called NMD670 in 81 adults with Charcot-Marie-Tooth disease types 1 and 2. Participants took either the drug or a placebo twice daily for 21 days. The main goal was to see if the drug helped them walk farther in a 6-minute test, and research…
Matched conditions: CMT
Phase: PHASE2 • Sponsor: NMD Pharma A/S • Aim: Symptom relief
Last updated May 11, 2026 20:48 UTC
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Gene sequencing breakthrough may unlock mysterious brain diseases in kids
Knowledge-focused CompletedThis study looked at whether whole genome sequencing (a complete scan of a person's DNA) can help doctors diagnose leukodystrophies—rare brain disorders that damage white matter—more accurately than standard methods. About 236 children with abnormal brain MRI scans but no known g…
Matched conditions: CMT
Sponsor: Children's Hospital of Philadelphia • Aim: Knowledge-focused
Last updated May 17, 2026 06:23 UTC