Study design & results
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Trial intelligence / Profile preview
RESPOND
2023-505640-18-00Phase 4Completed
- Sponsor
- Biogen
- Start
- Sep 2021
- 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
- 6
01
Overview
Phase 4, open-label trial of nusinersen in spinal muscular atrophy (SMA) (2 arms), sponsored by Biogen. Primary endpoint: Total Hammersmith Infant Neurological Examination (HINE) Section 2 Motor Milestones Score. Started Sep 2021; status: completed.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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Competitive context
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