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Japan Expanded Access Study of Tofersen for SOD1-ALS
jRCT2071240022Completed
- Sponsor
- Biogen
- Start
- Jun 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
- 1
01
Overview
Open-label, single-arm trial of tofersen in amyotrophic lateral sclerosis (ALS), sponsored by Biogen. Primary endpoint: Adverse event assessment. Started Jun 2024; status: completed.
02
Drugs
tofersen
03
Conditions
amyotrophic lateral sclerosis (ALS)
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