ASPIRO Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety And Efficacy Findings up to 72 Weeks Post-Treatment

被引：0

作者：

Shieh, Perry B. ^{[1
]}

Kuntz, Nancy ^{[2
]}

Smith, Barbara ^{[3
]}

Dowling, James ^{[4
]}

Mueller-Felber, Wolfgang ^{[5
]}

Boennemann, Carsten G. ^{[6
]}

Servais, Laurent ^{[7
]}

Muntoni, Francesco ^{[8
]}

Blaschek, Astrid ^{[5
]}

Neuhaus, Sarah ^{[6
]}

Alfano, Lindsay N. ^{[9
]}

Beggs, Alan H. ^{[10
]}

Buj-Bello, Ana ^{[11
]}

Childers, Martin ^{[12
]}

Duong, Tina ^{[13
]}

Graham, Robert J. ^{[10
]}

Jain, Minal ^{[14
]}

James, Emma S. ^{[15
]}

Lawlor, Michael W. ^{[16
]}

Lee, Jun ^{[15
]}

MacBean, Victoria ^{[17
]}

Mavilio, Fulvio ^{[15
]}

Murtagh, Michael ^{[15
]}

Noursalehi, Mojtaba ^{[15
]}

Prasad, Suyash ^{[15
]}

Rico, Salvador ^{[15
]}

机构：

[1] Univ Calif Los Angeles, Los Angeles, CA USA

[2] Ann & Robert H Lurie Childrens Hosp Chicago, Chicago, IL 60611 USA

[3] Univ Florida, Gainesville, FL 32611 USA

[4] Hosp Sick Children, Toronto, ON, Canada

[5] Klinikum Univ Munchen, Munich, Germany

[6] NIH Porter Neuroscience Res Ctr, Bethesda, MD USA

[7] Hop Armand Trousseau, Paris, France

[8] Great Ormond St Hosp Sick Children, London, England

[9] Nationwide Childrens Hosp, Columbus, OH USA

[10] Childrens Hosp Boston, Boston, MA USA

[11] Genethon, Evry, France

[12] AskBio, New York, NY USA

[13] Stanford Univ, Stanford, CA 94305 USA

[14] NIH Hatfield Clin Res Ctr, Bethesda, MD USA

[15] Audentes Therapeut, San Francisco, CA USA

[16] Med Coll Wisconsin, Milwaukee, WI USA

[17] Brunel Univ London, Uxbridge, Middx, England

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：

1053

引用

页数：5

共 23 条

[21] Efficacy of gene therapy for X-linked severe combined immunodeficiency: update on trial No.1 follow-up and preliminary results of multicentric collaborative trial No. 2 (Paris - Boston - London)
Hacein-Bey-Abina, S.
Caccaveli, L.
Touzot, F.
Dal-Cortivo, L.
Heritier, S.
Frange, P.
Thrasher, A.
Gaspar, B.
Baum, C.
Schambach, A.
Malik, P.
Van der Loo, H.
Williams, D.
Bushman, F.
de Saint-Basile, G.
Lim, A.
Debre, M.
Blanche, S.
Fischer, A.
Cavazzana-Calvo, M.
HUMAN GENE THERAPY, 2011, 22 (10) : A40 - A40
[22] Preliminary Safety and Efficacy Results from an Open-Label, Dose-Escalation Pilot Gene Therapy Study of FT-002 in Subjects with RPGR Gene Mutation Associated X-Linked Retinitis Pigmentosa (XLRP)
Wu, Jihong
Sui, Ruifang
MOLECULAR THERAPY, 2024, 32 (04) : 451 - 452
[23] The Guard1 clinical trial - A first in-human, phase 1/2 study evaluating AVR-RD-02, a hematopoietic stem cell (HSC) gene therapy for Gaucher disease: Preliminary safety, pharmacodynamic and clinical efficacy results from the subjects observed for up to 24 months post-infusion
Adera, Mathews
Veselinovic, Milena
Treohan, Anita
Golipour, Azadeh
Ridha, Essra
MOLECULAR GENETICS AND METABOLISM, 2023, 138 (02) : 3 - 4

← 1 2 3 →