Glycogen storage disease due to glucose-6-phosphatase deficiency type IA

MONDO:0009287

Glycogenosis due to glucose-6-phosphatase deficiency (G6P) type a, or glycogen storage disease (GSD) type 1a, is a type of glycogenosis due to G6P deficiency.

Also known as: G6P deficiency type 1a, G6PC glycogen storage disease, GSD due to G6P deficiency type 1a, GSD due to G6P deficiency type Ia, GSD type 1a, GSDIa, glycogen storage disease caused by mutation in G6PC, glycogen storage disease due to G6P deficiency type Ia

8 clinical trials for this condition and its sub-types, 7 tagged with Glycogen storage disease due to glucose-6-phosphatase deficiency type IA itself.

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Where it sits in the disease tree

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↑ Glycogen storage disease I (13)

Including sub-types (8) Tagged with Glycogen storage disease due to glucose-6-phosphatase deficiency type IA (7)

Trials to join now! 4 Not yet finished but already full! 1 Completed 2

New mRNA therapy aims to control blood sugar in rare genetic disease

Disease control Ongoing

This early-stage trial tests an mRNA drug called mRNA-3745 in 15 adults and children with glycogen storage disease type 1a (GSD1a), a rare genetic condition that causes dangerously low blood sugar. The drug is given by IV infusion and aims to help the body produce a missing enzym…

Phase 1/2 • Sponsor: ModernaTX, Inc. • Aim: Disease control

Last updated Jun 27, 2026 08:04 UTC

Glycogen storage disease due to glucose-6-phosphatase deficiency type IA

Where it sits in the disease tree

New mRNA therapy aims to control blood sugar in rare genetic disease