CMT
Clinical trials for CMT explained in plain language.
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Gene shot tested for rare nerve disease
Disease control CompletedThis early-stage study tested the safety of a new gene-based injection called Engensis in people with Charcot-Marie-Tooth disease type 1A (CMT1A), a genetic nerve disorder. Twelve adult participants received injections into their weakened leg muscles over 270 days. The main goal …
Matched conditions: CMT
Phase: PHASE1, PHASE2 • Sponsor: Helixmith Co., Ltd. • Aim: Disease control
Last updated Apr 01, 2026 14:42 UTC
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Gene test cuts diagnosis time for devastating childhood brain diseases from years to months
Diagnosis CompletedThis study tested whether using whole genome sequencing (a complete genetic map) as a first test could diagnose children with rare, inherited brain disorders called leukodystrophies faster and more accurately than the standard step-by-step approach. Researchers enrolled 236 child…
Matched conditions: CMT
Sponsor: Children's Hospital of Philadelphia • Aim: Diagnosis
Last updated Mar 25, 2026 14:07 UTC
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New drug tested for rare nerve disease that weakens muscles
Symptom relief CompletedThis study tested whether a new drug called NMD670 could help adults with Charcot-Marie-Tooth disease (CMT) walk farther and feel better. For 21 days, 81 participants with CMT took either the drug or a placebo pill twice daily. Researchers measured changes in walking distance, fa…
Matched conditions: CMT
Phase: PHASE2 • Sponsor: NMD Pharma A/S • Aim: Symptom relief
Last updated Mar 24, 2026 08:43 UTC