Details of the Drug

General Information of Drug (ID: DMHZ24N)

Drug Name

RP-A501

Indication

Disease Entry	ICD 11	Status	REF
Type 2 glycogen storage 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)

DTT Name	DTT ID	UniProt ID	MOA	REF
Lysosome-associated membrane glycoprotein 2 (LAMP2)	TTULDG7	LAMP2_HUMAN	Replacement	[1]

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Molecular Interaction Atlas (MIA)

Jump to Detail Molecular Interaction Atlas of This Drug

References

1	ClinicalTrials.gov (NCT03882437) Gene Therapy for Male Patients With Danon Disease (DD) Using RP-A501; AAV9.LAMP2B. U.S. National Institutes of Health.