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

[1] ASPIRO Gene therapy trial in X-linked myotubular myopathy (XLMTM): Update on preliminary safety and efficacy findings up to 72 weeks post-treatment
Shieh, P.
Kuntz, N.
Bonnemann, C.
Mueller-Felber, W.
Lawlor, M. W.
Servais, L.
Smith, B.
Noursalehi, M.
Prasad, S.
Dowling, J.
NEUROMUSCULAR DISORDERS, 2020, 30 : S65 - S66
[2] ASPIRO gene therapy trial in X-Linked Myotubular Myopathy (XLMTM): Update on preliminary efficacy and safety findings
Shieh, Perry
Kuntz, Nancy
Dowling, James
Mueller-Felber, Wolfgang
Bonnemann, Carsten
Foley, Dimah
Seferian, Andreea
Servais, Laurent
Lawlor, Michael
Noursalehi, Mojtaba
Prasad, Suyash
Rico, Salvador
Miller, Weston
GENETICS IN MEDICINE, 2022, 24 (03) : S350 - S350
[3] ASPIRO gene therapy trial in X-linked myotubular myopathy (XLMTM): update on preliminary efficacy and safety findings
Shieh, P.
Kuntz, N.
Dowling, J.
Mueller-Felber, W.
Blaschek, A.
Boennemann, C.
Foley, R.
Saade, D.
Seferian, A.
Servais, L.
Lawlor, M.
Noursalehi, M.
Prasad, S.
Rico, S.
Miller, W.
NEUROMUSCULAR DISORDERS, 2021, 31 : S47 - S47
[4] ASPIRO Phase 1/2 Gene Therapy Trial In X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety and Efficacy Findings
Shieh, Perry B.
Kuntz, Nancy
Smith, Barbara
Bonnemann, Carsten G.
Dowling, James J.
Lawlor, Michael W.
Mueller-Felber, Wolfgang
Noursalehi, Mo
Rico, Salvador
Servais, Laurent
Prasad, Suyash
MOLECULAR THERAPY, 2019, 27 (04) : 306 - 306
[5] Aspiro Phase 1/2 Gene Therapy Trial in X-Linked Myotubular Myopathy (Xlmtm): Preliminary Safety and Efficacy Findings
Kuntz, Nancy
Shieh, Perry B.
Smith, Barbara
Bonnemann, Carsten G.
Dowling, James J.
Lawlor, Michael W.
Mueller-Felber, Wolfgang
Noursalehi, Mo
Rico, Sal
Servais, Laurent
Prasad, Suyash
MOLECULAR THERAPY, 2018, 26 (05) : 4 - 4
[6] ASPIRO phase 1/2 gene therapy trial in X-linked motubular myopathy (XLMTM): update on preliminary safety and efficacy findings
Dowling, J.
Shieh, P.
Kuntz, N.
Bonnemann, C.
Muller-Felber, W.
Lawlor, M.
Servais, L.
Smith, B.
Noursalehi, M.
Rico, S.
Prasad, S.
NEUROMUSCULAR DISORDERS, 2019, 29 : S207 - S207
[7] Gene Therapy Trial in X-Linked Myotubular Myopathy (XLMTM): Update on Preliminary Safety, Efficacy and Pathology with AT132
Lawlor, Michael
Schoser, Benedikt
Sewry, Caroline
Margeta, Marta
Dowling, James
Bonnemann, Carsten
Kuntz, Nancy
Muller-Felber, Wolfgang
Noursalehi, Mo
Rico, Salvador
Servais, Laurent
Shieh, Perry
Smith, Barbara
Prasad, Suyash
JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY, 2019, 78 (06): : 522 - 523
[8] ASPIRO phase 1/2 gene therapy trial In X-linked myotubular myopathy: preliminary safety and efficacy findings
Kuntz, N.
Shieh, P.
Smith, B.
Bonnemann, C.
Dowling, J.
Lawlor, M.
Mueller-Felber, W.
Noursalehi, M.
Rico, S.
Servais, L.
Prasad, S.
NEUROMUSCULAR DISORDERS, 2018, 28 : S91 - S91
[9] Experiences of parents/caregivers of children in the ASPIRO X-Linked myotubular myopathy (XLMTM) gene therapy clinical trial: a qualitative study
Juando-Prats, C.
Hodwitz, K.
Kenneally, N.
Alfano, L.
Sarazen, M.
Coats, J.
NEUROMUSCULAR DISORDERS, 2023, 33 : S71 - S71
[10] Long term outcomes for X-Linked myotubular Myopathy (XLMTM) with gene replacement therapy, resamirigene bilparvovec: Preliminary results from ASPIRO
Shieh, P.
Kuntz, N.
Dowling, J.
Mueller-Felber, W.
Blaschek, A.
Bonnemann, C.
Foley, R.
Saade, D.
Seferian, A.
Servais, L.
Bowden, A.
Sarazen, M.
Coats, J.
Lakshman, N.
Han, C.
Prasad, S.
Rico, S.
Miller, W.
NEUROMUSCULAR DISORDERS, 2022, 32 : S44 - S44

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