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Trial intelligence / Profile preview
STELLAR-1: Salanersen Intrathecal Treatment in Presymptomatic Infants with Genetically Confirmed SMA
jRCT2031260161Recruiting
- Sponsor
- Biogen
- Start
- Jun 2026
- 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
- 2
01
Overview
Open-label, single-arm trial of salanersen in spinal muscular atrophy (SMA), sponsored by Biogen. Primary endpoints: Part 1: Percentage of Participants with 2 SMN2 Copies Sitting Without Support (for at Least 10 Seconds), Part 1: Percentage of Participants with 3 SMN2 Copies Walking Alone (for at Least 5 Steps) and 1 more. Started Jun 2026; status: recruiting.
02
Drugs
salanersen
03
Conditions
spinal muscular atrophy (SMA)
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