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Glycogen storage disease type 1a (GSD1a) research hub

1 indexed studies · 0 currently recruiting

ongoing, recruitingPhase I and Phase II (Integrated)- First administration to humans
A Phase 1/2, adaptive, open-label, single ascending dose to multiple ascending dose study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of mRNA-3745 in participants with glycogen storage disease type 1a (GSD1a), followed by an open-label extension
Spain · Poland · Netherlands