Skip to contents

  4. Wassenberg, Tessa; Molero-Luis, Marta; Jeltsch, Kathrin; Hoffmann, Georg F.; Assmann, Birgit; Blau, Nenad; Garcia-Cazorla, Angeles; Artuch, Rafael; Pons, Roser; Pearson, Toni S.; Leuzzi, Vincenco; Mastrangelo, Mario; Pearl, Phillip L.; Lee, Wang Tso; Kurian, Manju A.; Heales, Simon; Flint, Lisa; Verbeek, Marcel; Willemsen, Michèl; Opladen, Thomas

    Consensus guideline for the diagnosis and treatment of aromatic l-amino acid decarboxylase (AADC) deficiency

    Articles
    View online
    Schließen

    Access

    Close

    Bookmarks

    You can manage bookmarks using lists, please log in to your user account for this.

    Springer Science and Business Media LLC, 2017

    Published in: Orphanet Journal of Rare Diseases

  9. Kim, In-Ha; Chien, Yin-Hsiu; Hwu, Paul Wuh-Liang; Lee, Ni-Chung; Tseng, Sheng-Hong; Tai, Chun-Hwei

    Improved Motor Function in Children with AADC Deficiency Treated with Eladocagene Exuparvovec (PTC-AADC): Interim Findings from a Phase 2 Trial

    Conference Proceedings
    View online
    Schließen
    Close

    Bookmarks

    You can manage bookmarks using lists, please log in to your user account for this.

    Georg Thieme Verlag KG, 2021

    Published in: Abstracts of the 46th Annual Meeting of the Society for Neuropediatrics

  11. Hwu, Paul Wuh-Liang; Chien, Yin-Hsiu; Lee, Ni-Chung; Tseng, Sheng-Hong; Pykett, Mark; Tai, Chun-Hwei

    Improved Motor Function in Children With Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Treated With Eladocagene Exuparvovec (PTC-AADC): Interim Findings From a Phase 1/2 Study (2057)

    Articles
    View online
    Schließen

    Access

    Close

    Bookmarks

    You can manage bookmarks using lists, please log in to your user account for this.

    Ovid Technologies (Wolters Kluwer Health), 2021

    Published in: Neurology

  12. Chien, Yin-Hsiu; Hwu, Paul Wuh-Liang; Lee, Ni-Chung; Tseng, Sheng-Hong; Pykett, Mark; Tai, Chun-Hwei

    Improved Motor Function in Children With Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Treated With Eladocagene Exuparvovec (PTC-AADC): Interim Findings From a Phase 2 Trial (2366)

    Articles
    View online
    Schließen

    Access

    Close

    Bookmarks

    You can manage bookmarks using lists, please log in to your user account for this.

    Ovid Technologies (Wolters Kluwer Health), 2021

    Published in: Neurology

  13. Werner, Christian; Chien, Yin-Hsiu; Lee, Ni-Chung; Tseng, Sheng-Hong; Tai, Chun-Hwei; Hwu, Wuh-Liang; Conway, Anne Marie; Pykett, Mark

    AGIL-AADC Gene Therapy Results in Sustained Improvements in Motor and Developmental Milestones over 5 Years in Children with AADC Deficiency

    Conference Proceedings
    View online
    Schließen
    Close

    Bookmarks

    You can manage bookmarks using lists, please log in to your user account for this.

    Georg Thieme Verlag KG, 2019

    Published in: Neuropediatrics

  15. Chien, Yin-Hsiu; Lee, Ni-Chung; Tseng, Sheng-Hong; Tai, Chun-Hwei; Conway, Anne Marie; Gruis, Kirsten; Pykett, Mark; Hwu, Wuh-Liang

    Gene Therapy in Children with AADC Deficiency with AGIL-AADC Leads to De Novo Dopamine Production and Sustained Improvement in Motor Milestones over 5 Years (S29.008)

    Articles
    View online
    Schließen

    Access

    Close

    Bookmarks

    You can manage bookmarks using lists, please log in to your user account for this.

    Ovid Technologies (Wolters Kluwer Health), 2018

    Published in: Neurology