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

计划入组 15 人 · 首次发布于 2021 年 10 月 27 日

Glycogen Storage Disease Type Ia (GSDIa) Disease Monitoring Program

计划入组 140 人 · 首次发布于 2024 年 10 月 10 日

Overnight Feeding Study in Glycogen Storage Disease Type 1

计划入组 5 人 · 首次发布于 2013 年 10 月 11 日

glycogen synthase inhibitors (glycogen storage diseases)

Glycogen Storage Disease 1b (Prime Medicine)

mini-GDE gene therapy (AAV9, glycogen storage disease type III)

Self Storage Group Inc

United States

Rare Disease Research, LLC

CASTLEMAN DISEASE COLLABORATIVE NETWORK

United States

Beam Therapeutics' BEAM-101 Shows Promise in Sickle Cell Disease Trial

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Ultragenyx Completes BLA Submission for First Gene Therapy Targeting Glycogen Storage Disease Type Ia