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High-dose nusinersen versus approved dose in spinal muscular atrophy (Japan)
jRCT2061200040Completed
- Sponsor
- Biogen
- Start
- Aug 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
01
Overview
Randomised, double-blind trial of nusinersen in spinal muscular atrophy (SMA) (4 arms), sponsored by Biogen. Primary endpoints: Change from Baseline in CHOP INTEND Total Score (Part B infantile-onset SMA), Number of Participants with Adverse Events and Serious Adverse Events (Part A and C) and 18 more. Started Aug 2021; status: completed.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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