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Long-term extension study of Tofersen in adults with SOD1-associated amyotrophic lateral sclerosis
2016-003225-41Phase 3Completed
- Sponsor
- Biogen
- Start
- Apr 2017
- 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, open-label, single-arm trial of tofersen in amyotrophic lateral sclerosis (ALS) and familial amyotrophic lateral sclerosis (FALS), sponsored by Biogen. Primary endpoints: Number of participants experiencing adverse events and Number of participants experiencing serious adverse events. Started Apr 2017; status: completed.
02
Drugs
tofersen
03
Conditions
amyotrophic lateral sclerosis (ALS)familial amyotrophic lateral sclerosis (FALS)
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