Long-Term Outcomes of Eladocagene Exuparvovec Compared with Standard of Care in Aromatic l-Amino Acid Decarboxylase (AADC) Deficiency: A Modelling Study-Reference-Cited by-同舟云学术

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

Published:2023-10-06 Issue:12 Volume:40 Page:5399-5414
ISSN:0741-238X
Container-title:Advances in Therapy
language:en
Short-container-title:Adv Ther

Author:

Simons Claire L.,Hwu Wuh-Liang,Zhang Rongrong^ORCID,Simons Martijn J. H. G.,Bergkvist Mats,Bennison Craig

Funder

PTC Therapeutics

Publisher

Springer Science and Business Media LLC

Subject

Pharmacology (medical),General Medicine

Link

https://link.springer.com/content/pdf/10.1007/s12325-023-02689-6.pdf

Reference41 articles.

1. Wassenberg T, Molero-Luis M, Jeltsch K, et al. Consensus guideline for the diagnosis and treatment of aromatic l-amino acid decarboxylase (AADC) deficiency. Orphanet J Rare Dis. 2017;12(1):12.

2. Himmelreich N, Montioli R, Bertoldi M, et al. Aromatic amino acid decarboxylase deficiency: molecular and metabolic basis and therapeutic outlook. Mol Genet Metab. 2019;127(1):12–22.

3. Hyland K, Surtees RA, Rodeck C, Clayton PT. Aromatic l-amino acid decarboxylase deficiency: clinical features, diagnosis, and treatment of a new inborn error of neurotransmitter amine synthesis. Neurology. 1992;42(10):1980–8.

4. Hwu WL, Chien YH, Lee NC, Li MH. Natural history of aromatic l-amino acid decarboxylase deficiency in Taiwan. JIMD Rep. 2017;31(40):1–6.

5. Hwu WL, Muramatsu S, Tseng SH, et al. Gene therapy for aromatic l-amino acid decarboxylase deficiency. Sci Transl Med. 2012;4(134):13461.

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