Update on a Two-Part, International, Real-World, Observational Registry of Subjects with Aromatic L-Amino Acid Decarboxylase Deficiency (AADCd) ± Eladocagene Exuparvovec Treatment.

This article provides an update on a two-part international observational registry called AADCAware, which focuses on individuals with aromatic L-amino acid decarboxylase deficiency (AADCd). AADCd is a rare neurodevelopmental disorder that causes severe impairment. Part A of the registry examines the natural history of AADCd in patients receiving standard care, while Part B assesses the long-term safety and efficacy of a specific treatment called eladocagene exuparvovec on motor function. The study found that the majority of subjects failed to achieve major motor milestones, indicating a significant need for daily life support. [Extracted from the article]