Charcot-Marie-Tooth disease type 2
MONDO:0018993A Charcot-Marie-Tooth disease characterized by abnormalities in the axon of the peripheral nerve cell.
Also known as: CMT2, autosomal dominant axonal Charcot-Marie-Tooth disease, hereditary motor and sensory neuropathy type 2, Charcot-Marie-Tooth type 2, autosomal dominant Charcot-Marie-Tooth disease type 2
16 clinical trials for this condition and its sub-types.
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Broader categories
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One-Person trial aims to treat rare nerve disorder with custom drug
Disease control ENROLLING_BY_INVITATIONThis study tests a custom-made drug called an antisense oligonucleotide (ASO) designed for one person with Charcot-Marie-Tooth disease type 2D (CMT2D) caused by a specific GARS1 gene mutation. The drug aims to improve motor skills and quality of life. Only one participant is enro…
Phase: PHASE1, PHASE2 • Sponsor: n-Lorem Foundation • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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One-Patient trial hopes to fix rare nerve disease at its genetic root
Disease control OngoingThis study tests a custom-made drug (VCA-894A) in a single person with a rare genetic nerve disease called CMT2S. The drug is designed to correct a specific genetic error and restore a missing protein. The main goals are to check if the treatment is safe and if it can improve mus…
Phase: PHASE1, PHASE2 • Sponsor: Vanda Pharmaceuticals • Aim: Disease control
Last updated Jun 27, 2026 07:56 UTC
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One-Time gene injection aims to halt rare childhood paralysis
Disease control ENROLLING_BY_INVITATIONThis early-stage trial tests a single injection of gene therapy for people with SMARD1 or CMT2S, rare diseases caused by mutations in the IGHMBP2 gene. The therapy delivers a working copy of the gene directly into the spinal fluid. Ten participants, ranging from infants to childr…
Phase: PHASE1, PHASE2 • Sponsor: Megan Waldrop • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC