ELADOCAGENE EXUPARVOVEC GENE THERAPY IMPROVES MOTOR DEVELOPMENT IN PATIENTS WITH AROMATIC L-AMINO ACID DECARBOXYLASE DEFICIENCY

被引：0
|
作者：
Pachelli, P. E. [1 ]
Giugliani, L. [1 ]
Federhen, A. [1 ]
Wang, A. [2 ]
Russel, A. [2 ]
Roubertie, A. [3 ]
Chien, Y-H. [4 ]
Lee, N-C. [4 ]
Tai, C-H. [4 ]
Wuh-Liang, P. [4 ]
机构：
[1] PTC Farmaceut Brasil LTDA, Sao Paulo, Brazil
[2] PTC Therapeut, South Plainfield, NJ USA
[3] Univ Hosp Montpellier, Montpellier, France
[4] Natl Taiwan Univ Hosp, Taipei, Taiwan
来源：
CYTOTHERAPY | 2022年 / 24卷 / 10期
关键词：
Rare diseases; Genetics; Movement disorders (including cerebral palsy);
D O I：
暂无
中图分类号：
Q813 [细胞工程];
学科分类号：
摘要：
13
引用
收藏
页码：S5 / +
页数：2
相关论文
共 50 条
  • [1] Eladocagene Exuparvovec Gene Therapy Improves Motor Development in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency
    Hwu, P.
    Chien, Y.
    Lee, N.
    Tseng, S.
    Wang, A.
    Wang, J.
    Schilling, T.
    Ozdas, S.
    Tai, C.
    ANNALS OF NEUROLOGY, 2021, 90 : S94 - S95
  • [2] Eladocagene exuparvovec gene therapy improves motor development in patients with aromatic L-amino acid decarboxylase deficiency
    Hwu, P.
    Sierra, J.
    Chien, Y-H
    Lee, N-C
    Wang, A.
    Turna, J.
    Penematsa, V
    Golden, L.
    Tai, C-H
    ANNALS OF NEUROLOGY, 2023, 94 : S137 - S137
  • [3] Eladocagene Exuparvovec Gene Therapy Improves Motor Development in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency
    Hwu, Paul W-L
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Tseng, Shen-Hong
    Wang, Antonia
    Schilling, Traci
    Tai, Chun-Hwei
    NEUROLOGY, 2022, 98 (18)
  • [4] Eladocagene exuparvovec gene therapy improves motor development in patients with aromatic L-amino acid decarboxylase deficiency
    Warn, L.
    Chien, Y-H
    Lee, N-C
    Werner, C.
    Krolick, A.
    Wang, A.
    Turna, J.
    Hwu, Wuh-Liang P.
    Penematsa, V
    Golden, L.
    Tai, C-H
    ANNALS OF NEUROLOGY, 2025, 96 : S25 - S25
  • [5] Eladocagene Exuparvovec Gene Therapy Improves Motor Development in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency
    Russell, Alexis
    Roubertie, Agathe
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Sierra, Rafael
    Turna, Jasmine
    Wang, Antonia
    Tai, Chun-Hwei
    Hwu, Paul W-L
    NEUROLOGY, 2023, 100 (17)
  • [6] Eladocagene exuparvovec gene therapy improves motor development in patients with aromatic l-amino acid decarboxylase deficiency
    Hwu, P. W-L
    Chien, Y. H.
    Lee, N. C.
    Tseng, S. H.
    Wang, A.
    Wang, J.
    Leonardi, E. T.
    Schilling, T.
    Ozdas, S.
    Tai, C. H.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A44 - A44
  • [7] Gene Therapy With Eladocagene Exuparvovec Improves Cognition and Language in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency
    Hwu, P.
    Chien, Y.
    Lee, N.
    Tseng, S.
    Wang, A.
    Wang, J.
    Schilling, T.
    Ozdas, S.
    Tai, C.
    ANNALS OF NEUROLOGY, 2021, 90 : S98 - S99
  • [8] Gene therapy with eladocagene exuparvovec improves cognition and language in patients with aromatic l-amino acid decarboxylase deficiency
    Hwu, P. W-L
    Chien, Y. H.
    Lee, N. C.
    Tseng, S. H.
    Wang, A.
    Wang, J.
    Leonardi, E. T.
    Schilling, T.
    Ozdas, S.
    Tai, C. H.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A43 - A43
  • [9] Eladocagene Exuparvovec for Aromatic L-Amino Acid Decarboxylase Deficiency
    Golikeri, Avanti
    Yi, Sojeong
    Fashoyin-Aje, Lola
    JAMA-JOURNAL OF THE AMERICAN MEDICAL ASSOCIATION, 2025,
  • [10] Eladocagene Exuparvovec Improves Body Weight and Reduces Respiratory Infections in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency
    Hwu, P.
    Chien, Y.
    Lee, N.
    Tseng, S.
    Wang, A.
    Wang, J.
    Schilling, T.
    Ozdas, S.
    Tai, C.
    ANNALS OF NEUROLOGY, 2021, 90 : S100 - S100
12345