Mutation in the superoxide dismutase-1 (SOD1) gene

Clinical trials for Mutation in the superoxide dismutase-1 (SOD1) gene explained in plain language.

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New hope for ALS: first human trial of Gene-Targeting drug begins

Disease control Recruiting now

This study tests an experimental drug called ALN-SOD in people with ALS caused by a mutation in the SOD1 gene. The drug is given via spinal injection to target the root cause of the disease. The main goal is to check safety, but researchers will also measure effects on biomarkers…

Matched conditions: MUTATION IN THE SUPEROXIDE DISMUTASE-1 (SOD1) GENE

Phase 1/2 • Sponsor: Regeneron Pharmaceuticals • Aim: Disease control

Last updated Sep 13, 2026 00:00 UTC

Mutation in the superoxide dismutase-1 (SOD1) gene

Get alerted when new Mutation in the superoxide dismutase-1 (SOD1) gene trials appear

New hope for ALS: first human trial of Gene-Targeting drug begins