ArticleOrphanet journal of rare diseases2025

Clinically meaningful improvements after gene therapy for aromatic L-amino acid decarboxylase deficiency (AADCd) in the Peabody Developmental Motor Scale, Second Edition (PDMS-2) and correlation with Bayley-III scores and motor milestones.

Wuh-Liang Hwu et al.PubMed ↗Full text ↗Publisher ↗

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