Long-Term Outcomes of Eladocagene Exuparvovec Compared with Standard of Care in Aromatic l-Amino Acid Decarboxylase (AADC) Deficiency: A Modelling Study

INTRODUCTION: Aromatic l-amino acid decarboxylase (AADC) deficiency is a rare disease with symptoms including movement disorders, developmental delays, and autonomic symptoms starting from birth; further, patients with AADC deficiency are at a high risk of death in the first decade of life. Limited...

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Detalles Bibliográficos
Autores principales: Simons, Claire L., Hwu, Wuh-Liang, Zhang, Rongrong, Simons, Martijn J. H. G., Bergkvist, Mats, Bennison, Craig
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Springer Healthcare 2023
Materias:
Original Research
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10611606/
https://www.ncbi.nlm.nih.gov/pubmed/37803205
http://dx.doi.org/10.1007/s12325-023-02689-6

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