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Trial intelligence / Profile preview
First-in-human open-label oral LMI070 study in infants with type 1 spinal muscular atrophy
NL-OMON41741Phase 2Withdrawn
- Sponsor
- Novartis
- Start
- Nov 2014
- Modality
- Small Molecules
- Countries
- 1
01
Overview
Phase 2, open-label trial of branaplam in spinal muscular atrophy type 1 (2 arms), sponsored by Novartis. Primary endpoints: Safety and tolerability in Part 1, Safety and tolerability in Part 2 and 1 more. Planned start Nov 2014; status: withdrawn.
02
Drugs
branaplam
03
Conditions
spinal muscular atrophy type 1
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