Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial

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Publication Details

Journal
Journal of Neurology
Published
2025-08-16
DOI
https://doi.org/10.1007/s00415-025-13266-y
Citations
5
Primary Topic
Lysosomal Storage Disorders Research
Type
article
Field-Weighted Citation Impact
5.82

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article

Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial

Volker Straub, Mark Jonathan Roberts, Ans T. van der Ploeg, Gérson Carvalho et al.
5 citations
Journal of Neurology
Lysosomal Storage Disorders Research
5.82
article

Efficacy and safety of avalglucosidase alfa in patients with late-onset Pompe disease after 145 weeks of treatment during the COMET trial

Volker Straub, Mark Jonathan Roberts, Ans T. van der Ploeg, Gérson Carvalho, Swathi Tammireddy, Priya Sunil Kishnani, С. Н. Иллариошкин, Sevim Erdem‐Özdamar, Benedikt G. H. Schoser, Shafeeq Ladha, John Day, Magali Periquet, Mazen M. Dimachkie, Kristina An Haack, Hani Kushlaf, Özlem Göker-Alpan, Olivier Huynh‐Ba, Jordi Díaz‐Manera, Anna Kostera‐Pruszczyk, Shahram Attarian, António Toscano, Paula R. Clemens, Tahseen Mozaffar, Tianyue Zhou, Nathan Thibault
article en
5 citations

Abstract

No abstract available for this paper.
Journal of NeurologyVol. 272(9)
University of Messina (IT), Barrow Neurological Institute (US), University of Pittsburgh (US), University of California, Irvine (US), Medical University of Warsaw (PL), Veterans Health Administration (US), Salford Royal NHS Foundation Trust (GB), Newcastle upon Tyne Hospitals NHS Foundation Trust (GB), Erasmus MC (NL), Sanofi (France) (FR), LMU Klinikum (DE), Lysosomal and Rare Disorders Research and Treatment Center (US), Sanofi (United States) (US), Instituto de Pesca (BR), Duke Medical Center (US), University of Kansas Medical Center (US), Sanofi (Germany) (DE), Research Center of Neurology (RU), Hôpital de la Timone (FR), University of Cincinnati (US), Hacettepe University (TR), Ludwig-Maximilians-Universität München (DE), Newcastle University (GB), University of Cincinnati Medical Center (US), Stanford University (US)
U.S. Department of Veterans Affairs, Sanofi
Good health and well-being
Openalex Percentile: Top 4%
Lysosomal Storage Disorders Research
5.82
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