Charcot-Marie-Tooth disease type 4J
MONDO:0012640Charcot-Marie-Tooth disease, type 4J (CMT4J) belongs to the genetically heterogeneous group of CMT peripheral sensorimotor polyneuropathy diseases.
Also known as: CMT4J, Charcot-Marie-Tooth disease type 4 caused by mutation in FIG4, FIG4 Charcot-Marie-Tooth disease type 4, Charcot-Marie-Tooth disease, autosomal recessive, type 4J, Charcot-Marie-Tooth disease, type 4J
12 clinical trials for this condition and its sub-types, 3 tagged with Charcot-Marie-Tooth disease type 4J itself.
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One-Time gene shot aims to stop rare nerve disease in kids
Disease control Recruiting nowThis early-phase trial tests a single injection of gene therapy (ELP-02) into the spinal fluid of 8 children and young adults with CMT4J, a rare genetic nerve disease that causes muscle weakness. The therapy delivers a working copy of the FIG4 gene to nerve cells, potentially slo…
Phase 1/2 • Sponsor: Elpida Therapeutics SPC • Aim: Disease control
Last updated Sep 11, 2026 00:00 UTC
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New study aims to unravel nerve mysteries in rare diseases
Knowledge-focused Recruiting nowThis study looks at how nerves work in people with Charcot-Marie-Tooth disease and other nerve disorders like CIDP. Researchers will use special electrical tests and ultrasound to measure nerve and muscle changes. The goal is to better understand these conditions, not to test a n…
Sponsor: Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta • Aim: Knowledge-focused
Last updated Jun 27, 2026 11:00 UTC