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Long-Term High-Dose Nusinersen Extension in Spinal Muscular Atrophy (Study 302)
jRCT2031220035Completed
- Sponsor
- Biogen
- Start
- Aug 2022
- 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
Open-label trial of nusinersen in spinal muscular atrophy (SMA) (2 arms), sponsored by Biogen. Primary endpoints: Incidence of adverse events, Change in growth parameters and 6 more. Started Aug 2022; status: completed.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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