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Phase 3 study of intrathecal nusinersen in later-onset spinal muscular atrophy
2014-001947-18Phase 3Completed
- Sponsor
- Ionis Pharmaceuticals
- Start
- Dec 2014
- 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
- 1
01
Overview
Phase 3, randomised, double-blind trial of nusinersen in spinal muscular atrophy (SMA) (2 arms), sponsored by Ionis Pharmaceuticals. Primary endpoint: Change from baseline in Hammersmith Functional Motor Scale Expanded score. Started Dec 2014; status: completed.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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