Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency-Reference-Cited by-同舟云学术

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

Published:2022-02 Issue:2 Volume:30 Page:509-518
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

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

Funder

PTC Therapeutics Inc

National Research Program for Biopharmaceuticals

National Taiwan University Hospital

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference37 articles.

1. Aromatic amino acid decarboxylase deficiency: molecular and metabolic basis and therapeutic outlook;Himmelreich;Mol. Genet. Metab.,2019

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

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

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

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

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