Safety and Improved Efficacy Outcomes in Children AADC Deficiency Treated with Eladocagene Exuparvovec Gene Therapy: Results From Three Clinical Trials

被引：0
|
作者：
Hwu, Paul Wuh-Liang [1 ]
Chien, Yin-Hsiu [1 ]
Lee, Ni-Chung [1 ]
Tseng, Sheng-Hong [1 ]
Tai, Chun-Hwei [1 ]
Conway, Anne Marie [2 ]
Pykett, Mark [2 ]
机构：
[1] Natl Taiwan Univ Hosp, Taipei, Taiwan
[2] PTC Therapeut Inc, South Plainfield, NJ USA
来源：
NEUROLOGY | 2020年 / 94卷 / 15期
关键词：
D O I：
暂无
中图分类号：
R74 [神经病学与精神病学];
学科分类号：
摘要：
4688
引用
收藏
页数：3
相关论文
共 50 条
  • [1] SAFETY AND IMPROVED EFFICACY OUTCOMES IN CHILDREN WITH AADC DEFICIENCY TREATED WITH ELADOCAGENE EXUPARVOVEC GENE THERAPY: RESULTS FROM THREE CLINICAL TRIALS
    Hwu, P. W. L.
    Pachelli, P. E.
    Chien, Y. H.
    Lee, N. C.
    Tseng, S. H.
    Tai, C. H.
    Conway, A. M.
    Federhen, A.
    Giugliani, L.
    Felix, T.
    Pykett, M.
    CYTOTHERAPY, 2021, 23 (04) : 33 - 33
  • [2] Safety and Improved Efficacy Outcomes in Children with AADC Deficiency Treated with AGIL-AADC Gene Therapy: Results from Three Clinical Trials
    Hwu, P. W-L
    Chien, Y-H
    Lee, N-C
    Tseng, S-H
    Tai, C-H
    Conway, A.
    Berner, T.
    Pykett, M.
    ANNALS OF NEUROLOGY, 2019, 86 : S148 - S149
  • [3] Reductions in oculogyric crisis in children with aadc deficiency treated with eladocagene exuparvovec gene therapy- results from 3 clinical trials
    Hwu, Paul Wuh-Liang
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Tseng, Sheng-Hong
    Wang, Antonia
    Schilling, Traci
    Wang, Jim
    Kristensen, Allan
    Ozdas, Sunay
    Tai, Chun-Hwei
    EPILEPSIA, 2021, 62 : 163 - 163
  • [4] Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency
    Tai, Chun-Hwei
    Lee, Ni-Chung
    Chien, Yin-Hsiu
    Byrne, Barry J.
    Muramatsu, Shin-Ichi
    Tseng, Sheng-Hong
    Hwu, Wuh-Liang
    MOLECULAR THERAPY, 2022, 30 (02) : 509 - 518
  • [5] Improved Motor Function in Children with AADC Deficiency Treated with Eladocagene Exuparvovec (PTC-AADC): Compassionate Use Study
    Hwu, Paul Wuh-Liang
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Tseng, Sheng-Hong
    Tai, Chun-Hwei
    MOLECULAR THERAPY, 2020, 28 (04) : 270 - 271
  • [6] Improved Motor Function in Children with AADC Deficiency Treated with Eladocagene Exuparvovec (PTC-AADC): Interim Findings from a Phase 2 Trial
    Chien, Yin-Hsiu
    Hwu, Paul Wuh-Liang
    Lee, Ni-Chung
    Tseng, Sheng-Hong
    Tai, Chun-Hwei
    MOLECULAR THERAPY, 2020, 28 (04) : 560 - 560
  • [7] Improved Motor Function in Children with AADC Deficiency Treated with Eladocagene Exuparvovec (PTC-AADC): Interim Findings from a Phase 1/2 Study
    Hwu, Paul Wuh-Liang
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Tseng, Sheng-Hong
    Tai, Chun-Hwei
    MOLECULAR THERAPY, 2020, 28 (04) : 272 - 272
  • [8] Reductions in Oculogyric Crisis Duration and Frequency in Children With Aromatic L-Amino Acid Decarboxylase Deficiency Treated With Eladocagene Exuparvovec Gene Therapy: Results From 3 Clinical Trials
    Chien, Y.
    Hwu, P.
    Lee, N.
    Tseng, S.
    Wang, A.
    Schilling, T.
    Wang, J.
    Kristensen, A.
    Ozdas, S.
    Tai, C.
    ANNALS OF NEUROLOGY, 2021, 90 : S99 - S100
  • [9] Reductions in oculogyric crisis duration and frequency in children with aromatic l-amino acid decarboxylase deficiency treated with eladocagene exuparvovec gene therapy: results from 3 clinical trials
    Hwu, P. W-L
    Chien, Y. H.
    Lee, N. C.
    Tseng, S. H.
    Wang, A.
    Wang, J.
    Leonardi, E. T.
    Schilling, T.
    Kristensen, A.
    Ozdas, S.
    Tai, C. H.
    HUMAN GENE THERAPY, 2021, 32 (19-20) : A48 - A48
  • [10] 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)
12345