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Nusinersen versus risdiplam for respiratory outcomes in pediatric spinal muscular atrophy
IRCT20240111060676N1Phase 2Completed
- Sponsor
- Shahid Beheshti University of Medical Sciences
- Start
- Dec 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
- Countries
- 1
01
Overview
Phase 2, open-label trial of nusinersen and risdiplam in spinal muscular atrophy (SMA) (2 arms), sponsored by Shahid Beheshti University of Medical Sciences. Primary endpoints: Effect of nusinersen on oxygen saturation, Effect of nusinersen on PCO2 and 4 more. Started Dec 2022; status: completed.
02
Drugs
nusinersenrisdiplam
03
Conditions
spinal muscular atrophy (SMA)
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