RGX-121 gene therapy for severe Mucopolysaccharidosis Type II (MPS II): Interim neurodevelopmental outcomes and biomarker results

被引：0
|
作者：
Harmatz, Paul [1 ]
Escolar, Maria [2 ]
Ficicioglu, Can [3 ]
Giugliani, Roberto [4 ]
Cho, Yoonjin [5 ]
Phillips, Dawn [5 ]
Nevoret, Marie-Laure [5 ]
Falabella, Paulo [5 ]
机构：
[1] UCSF Benioff Childrens Hosp Oakland, Gastroenterol, Oakland, CA USA
[2] UPMC Childrens Hosp Pittsburgh, Pediat, Pittsburgh, PA USA
[3] Childrens Hosp Philadelphia, Pediat, Philadelphia, PA 19104 USA
[4] Univ Fed Rio Grande do Sul, Med Genet, Porto Alegre, RS, Brazil
[5] REGENXBIO, Rockville, MD USA
来源：
NEUROLOGY | 2021年 / 96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：
4164
引用
收藏
页数：4
相关论文
共 50 条
  • [1] RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): Interim results of an ongoing first in human trial
    Nevoret, Marie-Laure
    Escolar, Maria
    Ficicioglu, Can
    Giugliani, Roberto
    Harmatz, Paul
    Cho, Yoonjin
    Phillips, Dawn
    Falabella, Paulo
    MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S76 - S76
  • [2] RGX-121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II (MPS II): Interim Analysis of Data from the First in Human Study
    Ficicioglu, C.
    Escolar, M.
    Giugliani, R.
    Harmatz, P.
    Nevoret, M-L
    Fiscella, M.
    Yang, L.
    Cho, Y.
    Phillips, D.
    Falabella, P.
    ANNALS OF NEUROLOGY, 2021, 90 : S96 - S96
  • [3] RGX-121 gene therapy for the treatment of severe mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in-human study
    Harmatz, Paul
    Escolar, Maria
    Ficicioglu, Can
    Giugliani, Roberto
    Nevoret, Marie-Laure
    Fiscella, Michele
    Yang, Lin
    Cho, Yoonjin
    Phillipse, Dawn
    Falabella, Paulo
    MOLECULAR GENETICS AND METABOLISM, 2022, 135 (02) : S54 - S54
  • [4] RGX-121 gene therapy for the treatment of severe mucopolysaccharidosis type II (MPS II): Interim analysis of data from a Phase 1/2 study
    Harmatz, P.
    Giugliani, R.
    Escolar, M.
    Ficicioglu, C.
    Fiscella, M.
    Yang, L.
    Cho, Y.
    Falabella, P.
    ANNALS OF NEUROLOGY, 2022, 92 : S170 - S170
  • [5] RGX-121 GENE THERAPY FOR THE TREATMENT OF SEVERE MUCOPOLYSACCHARIDOSIS TYPE II (MPS II): INTERIM ANALYSIS OF DATA FROM A PHASE 1/2 STUDY
    Ficicioglu, Can
    Escolar, Maria
    Giugliani, Roberto
    Harmatz, Paul
    Nevoret, Marie-Laure
    Fiscella, Michele
    Yang, Lin
    Cho, Yoonjin
    Phillips, Dawn
    Falabella, Paulo
    MOLECULAR GENETICS AND METABOLISM, 2022, 135 (04) : 270 - 271
  • [6] RGX-121 Gene Therapy for the Treatment of Severe Mucopolysaccharidosis Type II: Interim Analysis of the First in Human Study
    Giugliani, Roberto
    Escolar, Maria
    Ficicioglu, Can
    Harmatz, Paul
    Nevoret, Marie-Laure
    Cho, Yoonjin
    Phillips, Dawn
    Falabella, Paulo
    MOLECULAR THERAPY, 2021, 29 (04) : 112 - 113
  • [7] RGX-121 gene therapy for severe mucopolysaccharidosis type II (MPS II): A clinical program to address central nervous system manifestations
    Cowley, Kirsten
    Falabella, Paulo
    Pakola, Stephen
    Nevoret, Marie-Laure
    MOLECULAR GENETICS AND METABOLISM, 2021, 132 (02) : S29 - S29
  • [8] RGX-121 Gene Therapy for the Treatment of Neuronopathic Mucopolysaccharidosis Type II (MPS II): Interim Analysis of Data from the First in Human Study
    Harmatz, Paul
    Ficicioglu, Can
    Giugliani, Roberto
    Rajan, Deepa
    Hagood, Joseph
    Fiscella, Michele
    Yang, Lin
    Gilmor, Michelle
    Cho, Yoonjin
    Phillips, Dawn
    Pisani, Laura
    Falabella, Paulo
    MOLECULAR THERAPY, 2023, 31 (04) : 398 - 398
  • [9] RGX-121 gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II): Interim analysis of data from the first in human study
    Harmatz, Paul
    Ficicioglu, Can H.
    Giugliani, Roberto
    Rajan, Deepa
    Hagood, Joseph
    Fiscella, Michele
    Yang, Lin
    Gilmor, Michelle
    Cho, Yoonjin
    Phillips, Dawn
    Falabella, Paulo
    MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 57 - 57
  • [10] CAMPSIITETM phase I/II/III: An interim clinical study update of RGX-121, an investigational gene therapy for the treatment of neuronopathic mucopolysaccharidosis type II (MPS II)
    Harmatz, Paul
    Ficicioglu, Can
    Giugliani, Roberto
    Rajan, Deepa
    Hagood, Joseph
    Fiscella, Michele
    Yang, Lin
    Gilmor, Michelle
    Cho, Yoonjin
    Mulatya, Caroline
    Phillips, Dawn
    Boulos, Nidal
    Falabella, Paulo
    Pisani, Laura
    MOLECULAR GENETICS AND METABOLISM, 2024, 141 (02)
12345