Study design & results
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Trial intelligence / Profile preview
Post-marketing open-label study of tofersen in Chinese adults with SOD1-ALS
CTR20253080Phase 4Active
- Sponsor
- Biogen
- Start
- Dec 2025
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
A Phase 4 trial of tofersen in amyotrophic lateral sclerosis (ALS), sponsored by Biogen. Started Dec 2025; status: active.
02
Drugs
tofersen
03
Conditions
amyotrophic lateral sclerosis (ALS)
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Competitive context
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