First-In-Human Intrathecal Gene Transfer Study for Giant Axonal Neuropathy: Three-Year Study Review of Safety, Immunologic Responses and Interim Analysis of Efficacy

被引：0
|
作者：
Bharucha-Goebel, D.
Saade, D.
Jain, M.
Waite, M.
Norato, G.
Cheung, K.
Foley, A. R.
Soldatos, A.
Rybin, D.
Lehky, T.
Del Hoyo, Calcedo R.
Jacobson, S.
Samulski, R. J.
Gray, S.
Bonnemann, C.
机构：
来源：
ANNALS OF NEUROLOGY | 2019年 / 86卷
关键词：
Neuromuscular Disorders; Translational/Experimental Therapeutics; Genetics;
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：
260
引用
收藏
页码：S168 / S168
页数：1
相关论文
共 24 条
  • [21] XC001 gene therapy for the treatment of refractory angina: 6-month efficacy and safety from the phase 1/2 first-in-human study (EXACT Trial)
    Stewart, D. J.
    Dittrich, H. C.
    Reinhardt, R. R.
    Rosengart, T. K.
    Crystal, R. G.
    Povsic, T. J.
    HUMAN GENE THERAPY, 2024, 35 (3-4) : A44 - A44
  • [22] Safety and Efficacy of Adct-402 (Loncastuximab Tesirine), a Novel Antibody Drug Conjugate, in Relapsed/Refractory Follicular Lymphoma and Mantle Cell Lymphoma: Interim Results from the Phase 1 First-in-Human Study
    Caimi, Paolo
    Kahl, Brad S.
    Hamadani, Mehdi
    Carlo-Stella, Carmelo
    He, Shui
    Ungar, David
    Feingold, Jay
    Ardeshna, Kirit M.
    Radford, John
    Solh, Melhem
    Heffner, Leonard
    O'Connor, Owen A.
    BLOOD, 2018, 132
  • [23] One-year initial efficacy and safety outcomes of the premounted dry-pericardium Vienna self-expandable transcatheter aortic valve system: A first-in-human VIVA feasibility study
    Briedis, Kasparas
    Rumbinaite, Egle
    Aldujeli, Ali
    Briede, Kamilija
    Jurenas, Martynas
    Jakuska, Povilas
    Jankauskas, Antanas
    Ceponiene, Indre
    Lenkutis, Tadas
    Plisiene, Jurgita
    Benetis, Rimantas
    Zaliunas, Remigijus
    CATHETERIZATION AND CARDIOVASCULAR INTERVENTIONS, 2024, 103 (07) : 1111 - 1124
  • [24] Interim analysis of first-in-human phase 1 study to assess safety and efficacy of YBL-006, an anti-PD-1 antibody in advanced solid tumor with exploratory biomarker analysis of tumor mutational burden and artificial intelligence (AI)-powered spatial analysis of tumor-infiltrating lymphocytes.
    Park, John J.
    Lee, Keun Wook
    Oh, Do-Youn
    Sabanathan, Dhanusha
    Kim, Se Hyun
    Kim, Tae Min
    Kim, Myungsuk
    Yoon, Jaebong
    Lee, Hyunmi
    Kim, Boram
    Ko, Yunjung
    Jeon, Eunyoung
    Cheon, Seonghye
    Shim, Eunyoung
    Park, Bum-Chan
    Lee, Han Seung
    Park, Seongyeol
    Paeng, Kyunghyun
    Ock, Chan-Young
    JOURNAL OF CLINICAL ONCOLOGY, 2021, 39 (15)
123