COMET: Efficacy and safety of avalglucosidase alfa in late-onset Pompe disease participants after 97 weeks of treatment

被引：0

作者：

Schoser, B. ^{[1
]}

Kishnani, P. ^{[2
]}

Diaz-Manera, J. ^{[3
,4
]}

Kushlaf, H. ^{[5
,6
]}

Ladha, S. ^{[7
]}

Mozaffar, T. ^{[8
]}

Straub, V. ^{[3
,4
]}

Toscano, A. ^{[9
]}

Van der Ploeg, A. ^{[10
]}

Berger, K. ^{[11
,12
]}

Clemens, P. ^{[13
,14
]}

Chien, Y. ^{[15
]}

Day, J. ^{[16
,17
]}

Ilarioshkin, S. ^{[18
]}

Roberts, M. ^{[19
]}

Attarian, S. ^{[20
]}

Carvalho, G. ^{[21
]}

Choi, Y. ^{[22
]}

Erdem-Ozdamar, S. ^{[23
]}

Goker-Alpan, O. ^{[24
]}

Kostera-Pruszczyk, A. ^{[25
]}

Haack, K. An ^{[26
]}

Thibault, N. ^{[27
]}

Zhou, T. ^{[27
]}

Dimachkie, M. ^{[28
]}

机构：

[1] LMU Klinikum Munchen, Dept Neurol, Munich, Germany

[2] Duke Univ, Med Ctr, Dept Pediat, Div Med Genet, Durham, NC 27710 USA

[3] Newcastle Univ, John Walton Muscular Dystrophy Res Ctr, Newcastle Upon Tyne, Tyne & Wear, England

[4] Newcastle Hosp NHS Fdn Trust, Newcastle Upon Tyne, Tyne & Wear, England

[5] Univ Cincinnati, Dept Neurol & Rehabil Med, Cincinnati, OH USA

[6] Univ Cincinnati, Dept Pathol & Lab Med, Cincinnati, OH USA

[7] Barrow Neurol Inst, Gregory W Fulton ALS & Neuromuscular Ctr, Phoenix, AZ 85013 USA

[8] Univ Calif Irvine, Dept Neurol, Orange, CA 92668 USA

[9] Univ Messina, Dept Clin & Expt Med, Reference Ctr Rare Neuromuscular Disorders, Messina, Italy

[10] Univ Med Ctr, Erasmus MC, Ctr Lysosomal & Metab Dis, Rotterdam, Netherlands

[11] NYU Grossman Sch Med, Div Pulm Crit Care & Sleep Med, New York, NY USA

[12] Bellevue Hosp, Andre Cournand Pulm Physiol Lab, New York, NY USA

[13] Univ Pittsburgh, Dept Neurol, Pittsburgh, PA 15260 USA

[14] Dept Vet Affairs Med Ctr, Pittsburgh, PA USA

[15] Natl Taiwan Univ Hosp, Dept Med Genet & Pediat, Taipei, Taiwan

[16] Stanford Univ, Dept Neurol, Stanford, CA 94305 USA

[17] Stanford Univ, Dept Pediat, Stanford, CA 94305 USA

[18] Res Ctr Neurol, Moscow, Russia

[19] Salford Royal NHS Fdn Trust, Salford, Lancs, England

[20] Hop La Timone, Referral Ctr Neuromuscular Dis & ALS, Marseille, France

[21] Inst Chronos Apoio Pesquisa, Brasilia, DF, Brazil

[22] Yonsei Univ, Gangnam Severance Hosp, Coll Med, Seoul, South Korea

[23] Hacettepe Univ, Dept Neurol, Fac Med, Ankara, Turkey

[24] Lysosomal & Rare Disorders Res & Treatment Ctr LD, Fairfax, VA USA

[25] Med Univ Warsaw, Dept Neurol, Warsaw, Poland

[26] Sanofi Genzyme, Chilly Mazarin, France

[27] Sanofi Genzyme, Cambridge, MA USA

[28] Univ Kansas, Med Ctr, Dept Neurol, Kansas City, KS 66103 USA

来源：

EUROPEAN JOURNAL OF NEUROLOGY | 2022年 / 29卷

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：

OPR-019

引用

页码：59 / 60

页数：2

共 50 条

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