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Presymptomatic SMA intrathecal ISIS 396443 open-label phase 2 study
2014-002098-12Phase 2Completed
- Sponsor
- Biogen
- Start
- Mar 2015
- 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 2, open-label, single-arm trial of tominersen in spinal muscular atrophy (SMA), sponsored by Biogen. Primary endpoint: Time to death or respiratory intervention. Started Mar 2015; status: completed.
02
Drugs
tominersen
03
Conditions
spinal muscular atrophy (SMA)
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