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Lysosomal glycogen storage disease

MONDO:0017738

67 clinical trials for this condition and its sub-types.

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Sub-types

Glycogen storage disease II (28) Glycogen storage disease due to acid maltase deficiency, late-onset (16) Glycogen storage disease due to acid maltase deficiency, infantile onset (9) Danon disease (5)

Broader categories

Disease (618) Metabolic disease (215) Hereditary disease (172) Inborn errors of metabolism (42) Lysosomal storage disease (33) Human disease (14) Disease of genetic or genomic mechanism (2) Disease by developmental or physiological process (0) Disease by etiologic mechanism (0)

Trials to join now! 35 Not yet recruiting 3 Not yet finished but already full! 15 Completed 13 Terminated 1

Pompe disease drug trial halted after just 3 patients

Disease control Terminated

This study tested an oral drug called duvoglustat in adults with Pompe disease, a rare genetic disorder that causes muscle weakness. Only 3 people took part, and the study was stopped early. Researchers looked at safety and whether the drug could improve walking distance and othe…

Phase: PHASE2 • Sponsor: Amicus Therapeutics • Aim: Disease control

Last updated Jun 26, 2026 16:43 UTC