Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial-Reference-Cited by-同舟云学术

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Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1/2 trial

Published:2017-12 Issue:4 Volume:1 Page:265-273
ISSN:2352-4642
Container-title:The Lancet Child & Adolescent Health
language:en
Short-container-title:The Lancet Child & Adolescent Health

Author:

Chien Yin-Hsiu,Lee Ni-Chung,Tseng Sheng-Hong,Tai Chun-Hwei,Muramatsu Shin-ichi,Byrne Barry J,Hwu Wuh-Liang

Publisher

Elsevier BV

Subject

Developmental and Educational Psychology,Pediatrics, Perinatology and Child Health

Reference26 articles.

1. Aromatic amino acid decarboxylase deficiency in twins;Hyland;J Inherit Metab Dis,1990

2. Clinical and biochemical features of aromatic L-amino acid decarboxylase deficiency;Brun;Neurology,2010

3. Aromatic L-amino acid decarboxylase deficiency: clinical features, treatment, and prognosis;Pons;Neurology,2004

4. Consensus guideline for the diagnosis and treatment of aromatic L-amino acid decarboxylase (AADC) deficiency;Wassenberg;Orphanet J Rare Dis,2017

5. 3-O-methyldopa levels in newborns: result of newborn screening for aromatic L-amino-acid decarboxylase deficiency;Chien;Mol Genet Metab,2016

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