CMT
Clinical trials for CMT explained in plain language.
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New injection shows promise for nerve disease safety
Disease control CompletedThis early-phase trial tested whether an experimental drug called Engensis (VM202) is safe when injected into the weakened leg muscles of people with Charcot-Marie-Tooth disease type 1A (CMT1A). Twelve adults with mild to moderate CMT1A received multiple injections over 270 days.…
Matched conditions: CMT
Phase: PHASE1, PHASE2 • Sponsor: Helixmith Co., Ltd. • Aim: Disease control
Last updated May 26, 2026 01:26 UTC
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New drug shows promise for nerve disease that weakens hands and feet
Symptom relief CompletedThis study tested an experimental drug called NMD670 in 81 adults with Charcot-Marie-Tooth disease types 1 and 2, a nerve condition that causes muscle weakness. Participants took either the drug or a placebo twice daily for 21 days. The main goal was to see if the drug helped the…
Matched conditions: CMT
Phase: PHASE2 • Sponsor: NMD Pharma A/S • Aim: Symptom relief
Last updated May 26, 2026 01:24 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 18, 2026 12:01 UTC