Abstract
Aromatic L-amino acid decarboxylase deficiency (AADCd) is a life-limiting and acutely debilitating rare genetic neurological disorder that causes widespread central nervous system dysfunction and developmental delay. Eladocagene exuparvovec is a gene therapy, approved in Europe, which bilaterally delivers a functional DDC gene directly into the putamen improving patient motor function. Due to the disease rarity and paucity of surrounding data, clinician experts were surveyed on evaluating the importance of motor development in AADC deficiency progression and outcomes.
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