Pompe disease treatment with twice a week high dose alglucoside alfa in a patient with severe dilated cardiomyopathy
Author:
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology
Reference11 articles.
1. Effects of a higher dose of alglucosidase alfa on ventilator-free survival and motor outcome in classic infantile Pompe disease: an open-label single-center study;van Gelder;J. Inherit. Metab. Dis.,2016
2. Improvement of bilateral ptosis on higher dose enzyme replacement therapy in Pompe disease;Yanovitch;J. Neuroophthalmol.,2010
3. Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease;Case;Neuromuscul. Disord.,2015
4. Enzyme replacement therapy with alglucosidase alfa in Pompe disease: clinical experience with rate escalation;Desai;Mol. Genet. Metab.,2018
5. Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease;Kishnani;Neurology,2007
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