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Children with aromatic l‐amino acid decarboxylase (AADC) deficiency suffer from severe motor dysfunction. Restoration of dopamine levels in the putamen by gene therapy has led to significant improvement in motor… Click to show full abstract
Children with aromatic l‐amino acid decarboxylase (AADC) deficiency suffer from severe motor dysfunction. Restoration of dopamine levels in the putamen by gene therapy has led to significant improvement in motor function. This study explored brain structure changes in patients.
Keywords:
acid decarboxylase;
aromatic amino;
amino acid;
gene therapy
Journal Title: Annals of Neurology
Year Published: 2019
Link to full text (if available)
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Journal Title: Annals of Neurology
Year Published: 2019
Link to full text (if available)
Share on Social Media: Sign Up to like & get
recommendations!