Drug Information
| Drug General Information | Top | |||
|---|---|---|---|---|
| Drug ID |
D0Q1UB
|
|||
| Former ID |
DIB005680
|
|||
| Drug Name |
GZ402666
|
|||
| Indication | Pompe disease [ICD-11: 5C51.3; ICD-10: E74.0] | Phase 1 | [1] | |
| Type 2 glycogen storage disease [ICD-11: 5C51.3; ICD-10: E74.0; ICD-9: 271] | Phase 1 | [2] | ||
| Target and Pathway | Top | |||
|---|---|---|---|---|
| Target(s) | Lysosomal alpha-glucosidase (GAA) | Target Info | Modulator | [1] |
| KEGG Pathway | Galactose metabolism | |||
| Starch and sucrose metabolism | ||||
| Metabolic pathways | ||||
| Lysosome | ||||
| Pathwhiz Pathway | Galactose Metabolism | |||
| Pathway Interaction Database | Notch-mediated HES/HEY network | |||
| References | Top | |||
|---|---|---|---|---|
| REF 1 | ClinicalTrials.gov (NCT01898364) Safety and Efficacy Evaluation of Repeat neoGAA Dosing in Late Onset Pompe Disease Patients.. U.S. National Institutes of Health. | |||
| REF 2 | Clinical pipeline report, company report or official report of the Pharmaceutical Research and Manufacturers of America (PhRMA) | |||
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