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ION363 intrathecal study in FUS-ALS
jRCT2031240101Phase 3Active, not recruiting
- Sponsor
- Ionis Pharmaceuticals
- Start
- Aug 2024
- Modality
- Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Single-strand DNA → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics, Modified DNA Oligonucleotides → Antisense DNA → DNA Therapeutics → Nucleic Acid Therapeutics
01
Overview
Phase 3 trial of ulefnersen in amyotrophic lateral sclerosis (ALS) and amyotrophic lateral sclerosis (fALS) (2 arms), sponsored by Ionis Pharmaceuticals. Primary endpoint: Change from baseline through Day 505 in functional impairment. Started Aug 2024; status: active, not recruiting.
02
Drugs
ulefnersen
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Conditions
amyotrophic lateral sclerosis (ALS)amyotrophic lateral sclerosis (fALS)
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