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Phase 3 BIIB067 in presymptomatic adults with SOD1 mutation
jRCT2071210032Phase 3Active, not recruiting
- Sponsor
- Biogen
- Start
- Aug 2021
- 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
01
Overview
Phase 3, randomised, double-blind trial of tofersen in amyotrophic lateral sclerosis (ALS) (7 arms), sponsored by Biogen. Primary endpoint: Emergence of clinically manifested ALS within 1 year of Part B baseline. Started Aug 2021; status: active, not recruiting.
02
Drugs
tofersen
03
Conditions
amyotrophic lateral sclerosis (ALS)
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