Transcriptomic characterization of clinical skeletal muscle biopsy from late-onset Pompe patients
Author:
Funder
Sanofi US
Publisher
Elsevier BV
Subject
Endocrinology,Genetics,Molecular Biology,Biochemistry,Endocrinology, Diabetes and Metabolism
Reference62 articles.
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2. The natural course of infantile Pompe’s disease: 20 original cases compared with 133 cases from the literature;van den Hout;Pediatrics,2003
3. A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease;Kishnani;J. Pediatr.,2006
4. Pompe’s disease;van der Ploeg;Lancet,2008
5. Recombinant human acid α-glucosidase: major clinical benefits in infantile-onset Pompe disease;Kishnani;Neurology,2007
Cited by 2 articles. 订阅此论文施引文献 订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献
1. Lentiviral gene therapy with IGF2-tagged GAA normalizes the skeletal muscle proteome in murine Pompe disease;Journal of Proteomics;2024-01
2. Omics-Based Approaches for the Characterization of Pompe Disease Metabolic Phenotypes;Biology;2023-08-23
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