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Functional effects of nusinersen in adults with 5q spinal muscular atrophy type 2 or 3
2020-005180-30Phase 4Completed
- Sponsor
- CHU de Reims
- Start
- Nov 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 4, randomised, single-blind trial of nusinersen in spinal muscular atrophy (SMA), spinal muscular atrophy type 2 (SMA2) and Spinal muscular atrophy type 3 (SMA3) (2 arms), sponsored by CHU de Reims. Primary endpoint: MFM-32 total score. Started Nov 2020; status: completed.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)spinal muscular atrophy type 2 (SMA2)Spinal muscular atrophy type 3 (SMA3)
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