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First human trial of ELP-02 aims to halt rare nerve disease in children

NCT ID NCT07447557

First seen Mar 16, 2026 · Last updated May 20, 2026 · Updated 13 times

Summary

This study tests a single dose of a new drug, ELP-02, given via a spinal injection to 8 people aged 3-20 with CMT4J, a rare genetic nerve disease that causes muscle weakness. The main goal is to check safety, but researchers will also see if it can slow or stop muscle damage. This is the first time the drug is being tested in humans.

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This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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