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Lysosomal glycogen storage disease

MONDO:0017738

45 clinical trials for this condition and its sub-types, 0 tagged with Lysosomal glycogen storage disease itself.

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Where it sits in the disease tree

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Part of

↑ Lysosomal storage disease (303)

Sub-types of Lysosomal glycogen storage disease

Glycogen storage disease II 31 trials · 41 incl. sub-types
2 sub-types
- Glycogen storage disease due to acid maltase deficiency, late-onset 19 trials
- Glycogen storage disease due to acid maltase deficiency, infantile onset 9 trials
Danon disease 5 trials

Including sub-types (45) Tagged with Lysosomal glycogen storage disease (0)

Trials to join now! 22 Not yet recruiting 2 Not yet finished but already full! 11 Completed 9 Terminated 1

Pompe disease drug trial halted after just 3 patients

Disease control Stopped early

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 2 • Sponsor: Amicus Therapeutics • Aim: Disease control

Last updated Jun 26, 2026 16:43 UTC