GENE TRANSFER

Clinical trials for GENE TRANSFER explained in plain language.

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Completed 1

Experimental gene therapy offers hope for rare nerve disease

Disease control Completed

This study tested a gene transfer treatment for giant axonal neuropathy (GAN), a rare genetic disease that damages nerves and affects movement, breathing, and other functions. Fourteen people aged 3 and older with GAN received the treatment through a spinal tap. The main goal was…

Matched conditions: GENE TRANSFER

Phase: PHASE1 • Sponsor: National Institute of Neurological Disorders and Stroke (NINDS) • Aim: Disease control

Last updated May 17, 2026 02:40 UTC

GENE TRANSFER

Get alerted when new GENE TRANSFER trials appear

Experimental gene therapy offers hope for rare nerve disease