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Higher Dose Nusinersen in Spinal Muscular Atrophy Patients Previously Treated With Risdiplam
2021-001294-23Phase 3Completed
- Sponsor
- Biogen
- Start
- Dec 2021
- 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 3, open-label, single-arm trial of nusinersen in spinal muscular atrophy (SMA), sponsored by Biogen. Primary endpoint: Change in Total Revised Upper Limb Module (RULM) Score. Started Dec 2021; status: completed.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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