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Dose-escalation study of intrathecal nusinersen in pediatric spinal muscular atrophy
2017-000327-27Phase 2
- Sponsor
- Ionis Pharmaceuticals
- Start
- Jan 2017
- 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 2, open-label, single-arm trial of nusinersen in spinal muscular atrophy (SMA), sponsored by Ionis Pharmaceuticals. Primary endpoint: Number of participants with adverse events and related measures.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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