Details of the Drug

General Information of Drug (ID: DM8WBNF)

Drug Name

DTX401

Indication

Disease Entry	ICD 11	Status	REF
Pompe disease	5C51.3	Phase 1	[1]
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Drug Type

Gene therapy

Cross-matching ID

Molecular Interaction Atlas of This Drug

Drug Therapeutic Target (DTT)

Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT03517085) Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults With Glycogen Storage Disease Type Ia (GSDIa). U.S. National Institutes of Health.
2	Clinical pipeline report, company report or official report of Ultragenyx Pharmaceutical.