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Nusinersen Effect on Ambulatory Function of Patients with Late-onset, Chronic SMA
jRCT1090220400Completed
- Sponsor
- Cyberdyne
- Start
- Dec 2018
- 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
Open-label, single-arm trial of nusinersen in spinal muscular atrophy (SMA), sponsored by Cyberdyne. Primary endpoint: 2-minute walk test. Started Dec 2018; status: completed.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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