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ATLAS presymptomatic SOD1-ALS tofersen trial
2023-505641-12-00Phase 3Authorised
- Sponsor
- Biogen
- Start
- Aug 2024
- 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
- 13
01
Overview
Phase 3, randomised, open-label trial of tofersen in amyotrophic lateral sclerosis (ALS) (4 arms), sponsored by Biogen. Primary endpoint: Percentage of participants with emergence of clinically manifest ALS within 24 months of Part B baseline. Planned start Aug 2024; status: authorised.
02
Drugs
tofersen
03
Conditions
amyotrophic lateral sclerosis (ALS)
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