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Nusinersen Pulmonary Function in SMA
NCT04050852Phase 1Withdrawn
- Sponsor
- NYU Langone Health
- Start
- Jul 2019
- Primary completion
- Apr 2021
- Modality
- Modified DNA Oligonucleotides, Single-strand DNA, Antisense Oligonucleotides (ASOs)
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of nusinersen in spinal muscular atrophy (SMA), sponsored by NYU Langone Health. Primary endpoints: Maximum Inspiratory Pressure at 12 months and Maximum Expiratory Pressure at 12 months. Planned start Jul 2019; status: withdrawn.
02
Drugs
nusinersen
03
Conditions
spinal muscular atrophy (SMA)
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