CHARCOT-MARIE-TOOTH DISEASE TYPE 4J

Clinical trials for CHARCOT-MARIE-TOOTH DISEASE TYPE 4J explained in plain language.

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Not yet recruiting 1

First-Ever gene therapy trial launches for devastating childhood nerve disease

Disease control Not yet recruiting

This is the first human study to test a single-dose gene therapy called ELP-02 for children and young adults (ages 3-20) with a rare, inherited nerve disease called Charcot-Marie-Tooth type 4J (CMT4J). The therapy aims to deliver a working copy of a faulty gene directly to the ne…

Matched conditions: CHARCOT-MARIE-TOOTH DISEASE TYPE 4J

Phase: PHASE1, PHASE2 • Sponsor: Elpida Therapeutics SPC • Aim: Disease control

Last updated Apr 01, 2026 14:41 UTC

CHARCOT-MARIE-TOOTH DISEASE TYPE 4J

Get alerted when new CHARCOT-MARIE-TOOTH DISEASE TYPE 4J trials appear

First-Ever gene therapy trial launches for devastating childhood nerve disease