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
  • [11] Clinical Improvements in the First Year Following Eladocagene Exuparvovec Gene Therapy in Patients With Aromatic L-Amino Acid Decarboxylase Deficiency
    Hwu, P.
    Wang, A.
    Russell, A.
    Turna, J.
    Trifillis, P.
    Tai, C-H
    ANNALS OF NEUROLOGY, 2022, 92 : S165 - S165
  • [12] Eladocagene exuparvovec improves body weight and reduces respiratory infections 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) : A2 - A2
  • [13] Eladocagene exuparvovec gene therapy increases Bayley-III cognitive and language raw scores in patients with aromatic L-amino acid decarboxylase deficiency
    Hwu, P.
    Lee, N-C
    Chien, Y-H
    Krolick, A.
    Sierra, R.
    Wang, A.
    Tai, C-H
    ANNALS OF NEUROLOGY, 2023, 94 : S93 - S93
  • [14] Gene therapy improves motor and mental function of aromatic l-amino acid decarboxylase deficiency
    Kojima, Karin
    Nakajima, Takeshi
    Taga, Naoyuki
    Miyauchi, Akihiko
    Kato, Mitsuhiro
    Matsumoto, Ayumi
    Ikeda, Takahiro
    Nakamura, Kazuyuki
    Kubota, Tetsuo
    Mizukami, Hiroaki
    Ono, Sayaka
    Onuki, Yoshiyuki
    Sato, Toshihiko
    Osaka, Hitoshi
    Muramatsu, Shin-ichi
    Yamagata, Takanori
    BRAIN, 2019, 142 : 322 - 333
  • [15] Gene Therapy for Aromatic L-Amino Acid Decarboxylase Deficiency
    Hwu, Wuh-Liang
    Muramatsu, Shin-ichi
    Tseng, Sheng-Hong
    Tzen, Kai-Yuan
    Lee, Ni-Chung
    Chien, Yin-Hsiu
    Snyder, Richard O.
    Byrne, Barry J.
    Tai, Chun-Hwei
    Wu, Ruey-Meei
    SCIENCE TRANSLATIONAL MEDICINE, 2012, 4 (134)
  • [16] Gene Therapy for Aromatic L-Amino Acid Decarboxylase Deficiency
    Zwagerman, Nathan T.
    Richardson, R. Mark
    NEUROSURGERY, 2012, 71 (04) : N10 - N12
  • [17] Gene therapy in the treatment of aromatic L-amino acid decarboxylase deficiency
    Kwiatkowska, Agnieszka
    Staniec, Magdalena
    Rocka, Agata
    Psiuk, Dominika
    Nowak, Emilia
    Filip, Agata
    PEDIATRIA I MEDYCYNA RODZINNA-PAEDIATRICS AND FAMILY MEDICINE, 2021, 17 (04): : 315 - 317
  • [18] Gene therapy improves brain white matter in aromatic l-amino acid decarboxylase deficiency
    Tseng, Chih-Hsien
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Hsu, Yung-Chin
    Peng, Shinn-Forng
    Tseng, Wen-Yih I.
    Hwu, Wuh-Liang
    ANNALS OF NEUROLOGY, 2019, 85 (05) : 644 - 652
  • [19] Improved Motor Function in Children With Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Treated With Eladocagene Exuparvovec (PTC-AADC): Compassionate Use Study
    Hwu, Paul Wuh-Liang
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Tseng, Sheng-Hong
    Pykett, Mark
    Tai, Chun-Hwei
    NEUROLOGY, 2021, 96 (15)
  • [20] Gene Therapy Improves Cerebral White Matter Microstructures in Patients with Aromatic L-Amino Acid Decarboxylase Deficiency
    Hwu, Wuh-Liang
    Lee, Ni-Chung
    Tseng, Chih-Hsien
    Chien, Yin-Hsiu
    Peng, Steven Shinn-Forng
    Tseng, Wen-Yih Isaac
    MOLECULAR THERAPY, 2018, 26 (05) : 31 - 31
12345