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Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency

Aromatic L-amino acid decarboxylase deficiency results in decreased neurotransmitter levels and severe motor dysfunction. Twenty-six patients without head control received bilateral intraputaminal infusions of a recombinant adeno-associated virus type 2 vector containing the human aromatic L-amino a...

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Detalles Bibliográficos
Autores principales:	Tai, Chun-Hwei, Lee, Ni-Chung, Chien, Yin-Hsiu, Byrne, Barry J., Muramatsu, Shin-Ichi, Tseng, Sheng-Hong, Hwu, Wuh-Liang
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2022
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8822132/ https://www.ncbi.nlm.nih.gov/pubmed/34763085 http://dx.doi.org/10.1016/j.ymthe.2021.11.005

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