Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study-Reference-Cited by-同舟云学术

Prospective exploratory muscle biopsy, imaging, and functional assessment in patients with late-onset Pompe disease treated with alglucosidase alfa: The EMBASSY Study

Published:2016-09 Issue:1-2 Volume:119 Page:115-123
ISSN:1096-7192
Container-title:Molecular Genetics and Metabolism
language:en
Short-container-title:Molecular Genetics and Metabolism

Author:

van der Ploeg Ans,Carlier Pierre G.,Carlier Robert-Yves,Kissel John T.,Schoser Benedikt,Wenninger Stephan,Pestronk Alan,Barohn Richard J.,Dimachkie Mazen M.,Goker-Alpan Ozlem,Mozaffar Tahseen,Pena Loren D.M.,Simmons Zachary,Straub Volker,Guglieri Michela,Young Peter,Boentert Matthias,Baudin Pierre-Yves,Wens Stephan,Shafi Raheel,Bjartmar Carl,Thurberg Beth L.

Funder

Sanofi Genzyme

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism

Reference42 articles.

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3. Glycogen storage disease type II: acid α-glucosidase (acid maltase) deficiency;Hirschhorn,2014

4. Pompe's disease;van der Ploeg;Lancet,2008

5. The stability of markers in dried-blood spots for recommended newborn screening disorders in the United States;Adam;Clin. Biochem.,2011

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1. Evaluating avalglucosidase alfa for the management of late-onset Pompe disease;Expert Review of Neurotherapeutics;2024-01-23

2. Безопасность и эффективность авалглюкозидазы альфа по сравнению с алглюкозидазой альфа у пациентов с болезнью Помпе с поздним началом: рандомизированное многоцентровое исследование III фазы COMET;Neuromuscular Diseases;2024-01-05

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5. Omics-Based Approaches for the Characterization of Pompe Disease Metabolic Phenotypes;Biology;2023-08-23