Eladocagene exuparvovec gene therapy improves motor development in patients with aromatic L-amino acid decarboxylase deficiency

被引：0
|
作者：
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
机构：
来源：
ANNALS OF NEUROLOGY | 2025年 / 96卷
关键词：
Neurodevelopmental Disorders;
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：
PL3-6
引用
收藏
页码：S25 / S25
页数：1
相关论文
共 50 条
  • [21] 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
  • [22] 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
  • [23] Long-Term Outcomes of Eladocagene Exuparvovec Compared with Standard of Care in Aromatic l-Amino Acid Decarboxylase (AADC) Deficiency: A Modelling Study
    Simons, Claire L.
    Hwu, Wuh-Liang
    Zhang, Rongrong
    Simons, Martijn J. H. G.
    Bergkvist, Mats
    Bennison, Craig
    ADVANCES IN THERAPY, 2023, 40 (12) : 5399 - 5414
  • [24] Long-Term Outcomes of Eladocagene Exuparvovec Compared with Standard of Care in Aromatic l-Amino Acid Decarboxylase (AADC) Deficiency: A Modelling Study
    Claire L. Simons
    Wuh-Liang Hwu
    Rongrong Zhang
    Martijn J. H. G. Simons
    Mats Bergkvist
    Craig Bennison
    Advances in Therapy, 2023, 40 : 5399 - 5414
  • [25] Gene Therapy for Aromatic L-Amino Acid Decarboxylase Deficiency: Report of Treatment for 8 Patients
    Hwu, Wuh-Liang
    Muramatsu, Shin-ichi
    Tseng, Sheng-Hong
    Tzen, Kai-Yuan
    Lee, Ni-Chung
    Chien, Yin-Hsiu
    Snyder, Richard O.
    Byrne, Barry
    Tai, Chun-Hwei
    Wu, Ruey-Meei
    MOLECULAR THERAPY, 2013, 21 : S25 - S25
  • [26] An Update on Gene Therapy for the Treatment of Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency
    Hwu, Wuh-Liang
    Muramatsu, Shin-Ichi
    Lee, Ni-Chung
    Chien, Yin-Hsiu
    Tseng, Sheng-Hong
    Tzen, Kai-Yuan
    Snyder, Richard O.
    Byrne, Barry J.
    Tai, Chun-Hwei
    Wu, Ruey-Meei
    MOLECULAR THERAPY, 2015, 23 : S103 - S103
  • [27] Intraputaminal Gene Delivery in Two Patients with Aromatic L-Amino Acid Decarboxylase Deficiency
    Francois-Heude, Marie-Celine
    Poulen, Gaetan
    Roze, Emmanuel Flamand
    Morel, Marie-Ange Nguyen
    Gras, Domitille
    Roch-Toreilles, Isabelle
    Quintard, Adeline
    Baroux, Gaelle
    Meyer, Pierre
    Coubes, Philippe
    Milesi, Christophe
    Cambonie, Gilles
    Baleine, Julien
    Sola, Chrystelle
    Delye, Benedicte
    Dimopoulou, Evgenia
    Sanchez, Stephanie
    Gasnier, Mathieu
    Touati, Souad
    Zamora, Alberto
    Pontal, Daniel
    Leboucq, Nicolas
    Kouyoumdjian, Virginie
    Lebasnier, Adrien
    Sanquer, Sylvia
    Mariano-Goulart, Denis
    Roujeau, Thomas
    Roubertie, Agathe
    MOVEMENT DISORDERS CLINICAL PRACTICE, 2023, 10 (05): : 811 - 818
  • [28] Aromatic L-amino acid decarboxylase deficiency in Taiwan
    Lee, Hsiu-Fen
    Tsai, Chi-Ren
    Chi, Ching-Shiang
    Chang, Tung-Ming
    Lee, Huei-Jane
    EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY, 2009, 13 (02) : 135 - 140
  • [29] 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
    Hwu, Paul Wuh-Liang
    Chien, Yin-Hsiu
    Lee, Ni-Chung
    Tseng, Sheng-Hong
    Pykett, Mark
    Tai, Chun-Hwei
    NEUROLOGY, 2021, 96 (15)
  • [30] FDA approves L-amino acid decarboxylase deficiency gene therapy
    Mullard, Asher
    NATURE REVIEWS DRUG DISCOVERY, 2025, 24 (01) : 6 - 6
12345