The effectiveness of enzyme replacement therapy for juvenile‐onset Pompe disease: A systematic review
Author:
Affiliation:
1. Adelaide Health Technology AssessmentUniversity of Adelaide Adelaide, Level 9, AHMS Building, North Terrace 5005 South Australia Australia
Funder
Department of Health, Australian Government
Publisher
Wiley
Subject
Genetics(clinical),Genetics
Link
https://onlinelibrary.wiley.com/doi/pdf/10.1002/jimd.12027
Reference38 articles.
1. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease
2. Enzyme replacement therapy in late-onset Pompe disease: a systematic literature review
3. Management and treatment of glycogenosis type II
4. Phenotype Variations in Early Onset Pompe Disease: Diagnosis and Treatment Results with Myozyme®
5. The natural course of non–classic Pompe’s disease; a review of 225 published cases
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3. Preclinical lentiviral vector-mediated hematopoietic stem and progenitor cell gene therapy corrects Pompe disease-related muscle and neurological manifestations;2023-11-05
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