Charcot-Marie-Tooth disease type 4
MONDO:0018995Charcot-Marie-Tooth disease type 4 (CMT4) belongs to the genetically heterogeneous group of CMT peripheral sensorimotor polyneuropathy diseases.
Also known as: AR-CMT1, CMT4, autosomal recessive demyelinating Charcot-Marie-Tooth
12 clinical trials for this condition and its sub-types.
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Broader categories
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One-Time gene shot aims to stop rare nerve disease in kids
Disease control Not yet recruitingThis 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: PHASE1, PHASE2 • Sponsor: Elpida Therapeutics SPC • Aim: Disease control
Last updated Jun 27, 2026 09:09 UTC
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Gene therapy readiness check: new study maps virus immunity in muscle disease patients
Knowledge-focused Not yet recruitingThis study will test blood samples from 450 people aged 6 to 60 with inherited neuromuscular diseases to see if they have antibodies that could block gene therapy viruses (AAVs). The goal is to understand how common these antibodies are and which patients might be good candidates…
Phase: NA • Sponsor: Genethon • Aim: Knowledge-focused
Last updated Jun 27, 2026 13:04 UTC