Study design & results
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Trial intelligence / Profile preview
ONWARD Long-Term Extension of Higher-Dose Nusinersen (Spinraza) in Spinal Muscular Atrophy
2023-505637-27-00Phase 3Completed
- Sponsor
- Biogen
- Start
- Jun 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
- Countries
- 15
01
Overview
Phase 3, open-label, single-arm trial of nusinersen in spinal muscular atrophy (SMA), sponsored by Biogen. Primary endpoints: Number of Participants With Adverse Events (AEs) and Serious Adverse Events (SAEs), Change from Baseline in Growth Parameters and 11 more. Started Jun 2024; status: completed.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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Competitive context
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