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Tofersen in presymptomatic adults with SOD1 mutation and elevated neurofilament
2020-004590-51Phase 3Active
- Sponsor
- Biogen
- Start
- Mar 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
- Countries
- 1
01
Overview
Phase 3, randomised, open-label trial of tofersen in amyotrophic lateral sclerosis (ALS) and familial amyotrophic lateral sclerosis (FALS) (4 arms), sponsored by Biogen. Primary endpoint: Percentage of participants with emergence of clinically manifested ALS within 24 months of Part B baseline. Started Mar 2021; status: active.
02
Drugs
tofersen
03
Conditions
amyotrophic lateral sclerosis (ALS)familial amyotrophic lateral sclerosis (FALS)
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