General Information of Drug (ID: DM9ZHE3)

Drug Name
PTC-AADC Drug Info
Indication
Disease Entry ICD 11 Status REF
Aromatic L-amino acid decarboxylase deficiency 5C59.00 Phase 2 [1]
Cross-matching ID
TTD Drug ID
DM9ZHE3

Molecule(s) Related to This Drug


Drug Therapeutic Target (DTT)
DTT Name DTT ID UniProt ID MOA REF
Aromatic-L-amino-acid decarboxylase (DDC) TTN451K DDC_HUMAN Not Available [1]

The Expression Level of Molecule(s) in Normal Tissue of Major ADME-Related Organs

Molecule Molecule Type Gene Name Liver Colon Kidney Small Intestine
Aromatic-L-amino-acid decarboxylase (DDC) DTT DDC 7.255 7.312 8.596 9.191
Molecule Expression Atlas in Normal Tissue of Major ADME-related organs

The Expression Level of Molecule(s) between Disease Section and Healthy Individual Tissue

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 between Disease Section and Healthy Individual Tissue

References

1 ClinicalTrials.gov (NCT04903288) An Open-Label Trial to Address the Safety of the SmartFlow MR-Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric Subjects. U.S.National Institutes of Health.