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Solid Biosciences GX1001: A randomized, controlled, open-label, single-ascending dose, phase I/II study to investigate the safety and tolerability, and efficacy of intravenous SGT-001 in male adolescents and childrenwith Duchenne muscular dystrophy.
Kuntz, Nancy L
(PD/PI)
Pediatrics
Project
:
Research project
Overview
Project Details
Status
Active
Effective start/end date
3/20/19
→
8/31/50
Funding
Solid Biosciences LLC
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