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Risdiplam pharmacokinetics and safety study in newborn infants with spinal muscular atrophy
NL-OMON58916Phase 2Completed
- Sponsor
- Roche
- Start
- Sep 2024
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of risdiplam in spinal muscular atrophy (SMA), sponsored by Roche. Primary endpoints: Plasma concentration of risdiplam and metabolites, AUC and 10 more. Started Sep 2024; status: completed.
02
Drugs
risdiplam
03
Conditions
spinal muscular atrophy (SMA)
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