Amyotrophic lateral sclerosis associated with A SOD1 gene mutation

Clinical trials for Amyotrophic lateral sclerosis associated with A SOD1 gene mutation explained in plain language.

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Can a shot stop ALS before it starts? new trial tests tofersen in gene carriers

Disease control Ongoing

This phase 3 trial tests whether the drug tofersen can delay or prevent ALS in adults who carry a SOD1 gene mutation but have no symptoms yet. About 158 participants will receive either tofersen or a placebo, and researchers will track how many develop ALS within two years. The g…

Matched conditions: AMYOTROPHIC LATERAL SCLEROSIS ASSOCIATED WITH A SOD1 GENE MUTATION

Phase 3 • Sponsor: Biogen • Aim: Disease control

Last updated Jun 27, 2026 12:02 UTC

Amyotrophic lateral sclerosis associated with A SOD1 gene mutation

Get alerted when new Amyotrophic lateral sclerosis associated with A SOD1 gene mutation trials appear

Can a shot stop ALS before it starts? new trial tests tofersen in gene carriers