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Higher-Dose Nusinersen in Patients With Spinal Muscular Atrophy Previously Treated With Risdiplam
jRCT2051260095Phase 3Active, not recruiting
- Sponsor
- Biogen
- Start
- May 2024
- 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
01
Overview
Phase 3, open-label, single-arm trial of nusinersen in spinal muscular atrophy (SMA), sponsored by Biogen. Primary endpoint: Change in total RULM score. Started May 2024; status: active, not recruiting.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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