GENE TRANSFER

Clinical trials for GENE TRANSFER explained in plain language.

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First human trial offers hope for rare, fatal nerve disease

Disease control Ongoing

This is a first-in-human safety study testing a new gene therapy for Giant Axonal Neuropathy (GAN), a rare and fatal genetic disorder that damages nerves. Researchers are giving a single spinal injection that delivers a working copy of the faulty gene to the brain and spinal cord…

Matched conditions: GENE TRANSFER

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

Last updated Mar 25, 2026 14:07 UTC

GENE TRANSFER

Get alerted when new GENE TRANSFER trials appear

First human trial offers hope for rare, fatal nerve disease