A position statement on the post gene-therapy rehabilitation of aromatic I-amino acid decarboxylase deficiency patients

A position statement on the post gene-therapy rehabilitation of aromatic I-amino acid decarboxylase deficiency patients

Abstract Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder of monoamine neurotransmitter synthesis that presents with a range of symptoms, including motor dysfunction and limited attainment of developmental motor milestones. The approval of eladocagene exuparvovec, a g...

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Bibliographic Details
Main Authors: Hui-Min Lee, Saadet Mercimek-Andrews, Gabriella Horvath, Diana Marchese, Richard E. Poulin, Alexis Krolick, Kati-Lyn Tierney, Jasmine Turna, Judy Wei, Wuh-Liang Hwu
Format: Article
Language:English
Published: BMC 2024-01-01
Series:Orphanet Journal of Rare Diseases
Subjects:
Gene therapy
AADC deficiency
Physical therapy
Neurotransmitter disorder
Rehabilitation outcomes
Gross motor development
Online Access:https://doi.org/10.1186/s13023-024-03019-x

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