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Intrathecal ISIS 396443 in Infantile-onset Spinal Muscular Atrophy
2013-004422-29Phase 3Completed
- Sponsor
- Ionis Pharmaceuticals
- Start
- Jul 2014
- Modality
- Small Interfering RNA (siRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, MicroRNA (miRNA) → Small RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics, Antisense Oligonucleotides (ASOs) → Long RNA Therapeutics → RNA Therapeutics → Nucleic Acid Therapeutics
- Countries
- 1
01
Overview
Phase 3, randomised, double-blind trial of tominersen in spinal muscular atrophy (SMA) (2 arms), sponsored by Ionis Pharmaceuticals. Primary endpoint: Time to death or permanent ventilation. Started Jul 2014; status: completed.
02
Drugs
tominersen
03
Conditions
spinal muscular atrophy (SMA)
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