Patient selection considerations for AADC deficiency gene therapy
Patient selection considerations for AADC deficiency gene therapy
About this item
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Publisher
Oxford: John Wiley & Sons, Inc
Journal title
Language
English
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Publisher
Oxford: John Wiley & Sons, Inc
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Contents
Background
Aromatic ʟ‐amino acid decarboxylase (AADC) deficiency is a rare, severe neurological disorder caused by pathogenic variants in the dopa decarboxylase (DDC) gene, resulting in a combined deficiency of monoamine neurotransmitters. Clinically, patients present with a range of dysfunctions that impact motor, autonomic, and cognitive devel...
Alternative Titles
Full title
Patient selection considerations for AADC deficiency gene therapy
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Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_deca6b443a2e40fb99fb7e172d08a585
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_deca6b443a2e40fb99fb7e172d08a585
Other Identifiers
ISSN
2831-3267,1755-5930
E-ISSN
2831-3267,1755-5949
DOI
10.1002/cns3.20052
