Details of the Drug

General Information of Drug (ID: DM8N3OQ)

Drug Name

GT-AADC

Indication

Disease Entry	ICD 11	Status	REF
Aromatic L-amino acid decarboxylase deficiency	5C59.00	Phase 2	[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
Aromatic-L-amino-acid decarboxylase (DDC)	TTN451K	DDC_HUMAN	Replacement	[1]

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Molecular Interaction Atlas (MIA)

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Molecular Expression Atlas of This Drug

The Studied Disease

Aromatic L-amino acid decarboxylase deficiency

ICD Disease Classification

5C59.00

Molecule Name	Molecule Type	Gene Name	p-value	Fold-Change	Z-score
Aromatic-L-amino-acid decarboxylase (DDC)	DTT	DDC	7.84E-03	-1.49	-1.01

Molecular Expression Atlas (MEA)

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References

1	Clinical pipeline report, company report or official report of PTC Therapeutics.