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Trial intelligence / Profile preview
Higher-dose intrathecal nusinersen study in spinal muscular atrophy
2019-002663-10Phase 2Completed
- Sponsor
- Biogen
- Start
- Feb 2020
- 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 2, randomised trial of nusinersen in spinal muscular atrophy (SMA) (3 arms), sponsored by Biogen. Primary endpoints: Safety and tolerability, Clinical efficacy measured by CHOP and 1 more. Started Feb 2020; status: completed.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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